Document and Entity Information
Document and Entity Information - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2018 | Mar. 04, 2019 | Jun. 30, 2018 | |
Document And Entity Information [Abstract] | |||
Document Type | 10-K | ||
Amendment Flag | false | ||
Document Period End Date | Dec. 31, 2018 | ||
Document Fiscal Year Focus | 2018 | ||
Document Fiscal Period Focus | FY | ||
Trading Symbol | DRNA | ||
Entity Registrant Name | Dicerna Pharmaceuticals Inc | ||
Entity Central Index Key | 0001399529 | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Well-known Seasoned Issuer | No | ||
Entity Current Reporting Status | Yes | ||
Entity Voluntary Filers | No | ||
Entity Filer Category | Accelerated Filer | ||
Entity Small Business | true | ||
Entity Emerging Growth Company | true | ||
Entity Shell Company | false | ||
Entity Ex Transition Period | true | ||
Entity Common Stock, Shares Outstanding | 68,264,949 | ||
Entity Public Float | $ 442.8 |
CONSOLIDATED BALANCE SHEETS
CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
CURRENT ASSETS: | ||
Cash and cash equivalents | $ 54,239 | $ 68,789 |
Held-to-maturity investments | 248,387 | 44,889 |
Contract receivables | 100,000 | 0 |
Withholding tax receivable | 0 | 1,583 |
Prepaid expenses and other current assets | 2,888 | 3,415 |
Total current assets | 405,514 | 118,676 |
NONCURRENT ASSETS: | ||
Property and equipment, net | 2,718 | 1,512 |
Restricted cash equivalents | 744 | 744 |
Other noncurrent assets | 65 | 70 |
Total noncurrent assets | 3,527 | 2,326 |
TOTAL ASSETS | 409,041 | 121,002 |
CURRENT LIABILITIES: | ||
Accounts payable | 5,013 | 4,920 |
Accrued expenses and other current liabilities | 9,649 | 5,726 |
Litigation settlement payable | 10,500 | 0 |
Current portion of deferred revenue | 68,893 | 6,180 |
Total current liabilities | 94,055 | 16,826 |
NONCURRENT LIABILITIES: | ||
Deferred revenue, net of current portion | 114,293 | 3,090 |
Total noncurrent liabilities | 114,293 | 3,090 |
TOTAL LIABILITIES | 208,348 | 19,916 |
COMMITMENTS AND CONTINGENCIES (NOTE 14) | ||
STOCKHOLDERS’ EQUITY: | ||
Preferred stock, $0.0001 par value – 5,000,000 shares authorized; no shares issued or outstanding at December 31, 2018 or 2017 | 0 | 0 |
Common stock, $0.0001 par value – 150,000,000 shares authorized; 68,210,742 and 51,644,841 shares issued and outstanding at December 31, 2018 and 2017, respectively | 7 | 5 |
Additional paid-in capital | 605,495 | 417,037 |
Accumulated deficit | (404,809) | (315,956) |
Total stockholders’ equity | 200,693 | 101,086 |
TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY | $ 409,041 | $ 121,002 |
CONSOLIDATED BALANCE SHEETS (Pa
CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares | Dec. 31, 2018 | Dec. 31, 2017 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized (in shares) | 5,000,000 | 5,000,000 |
Preferred stock, shares issued (in shares) | 0 | 0 |
Preferred stock, shares outstanding (in shares) | 0 | 0 |
Common stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized (in shares) | 150,000,000 | 150,000,000 |
Common stock, shares issued (in shares) | 68,210,742 | 51,644,841 |
Common stock, shares outstanding (in shares) | 68,210,742 | 51,644,841 |
CONSOLIDATED STATEMENTS OF OPER
CONSOLIDATED STATEMENTS OF OPERATIONS - USD ($) | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Income Statement [Abstract] | |||
Revenue from collaborative arrangements | $ 6,176,000 | $ 1,030,000 | $ 0 |
Operating expenses: | |||
Research and development | 45,711,000 | 35,888,000 | 41,399,000 |
General and administrative | 21,685,000 | 16,838,000 | 15,433,000 |
Litigation expense | 29,132,000 | 9,043,000 | 2,916,000 |
Total operating expenses | 96,528,000 | 61,769,000 | 59,748,000 |
Loss from operations | (90,352,000) | (60,739,000) | (59,748,000) |
Other income (expense): | |||
Interest income | 2,102,000 | 539,000 | 235,000 |
Interest expense | (603,000) | 0 | 0 |
Total other income, net | 1,499,000 | 539,000 | 235,000 |
Net loss | (88,853,000) | (60,200,000) | (59,513,000) |
Dividends on redeemable convertible preferred stock | 0 | (10,111,000) | 0 |
Deemed dividend related to beneficial conversion feature of redeemable convertible preferred stock | 0 | (6,144,000) | 0 |
Deemed dividend on conversion of redeemable convertible preferred stock | 0 | 3,837,000 | 0 |
Net loss attributable to common stockholders | $ (88,853,000) | $ (80,292,000) | $ (59,513,000) |
Net loss per share attributable to common stockholders - basic and diluted (in dollars per share) | $ (1.60) | $ (3.66) | $ (2.87) |
Weighted average common shares outstanding—basic and diluted (in shares) | 55,616,092 | 21,917,415 | 20,719,761 |
CONSOLIDATED STATEMENTS OF CHAN
CONSOLIDATED STATEMENTS OF CHANGES IN REDEEMABLE CONVERTIBLE PREFERRED STOCK AND STOCKHOLDERS' EQUITY - USD ($) $ in Thousands | Total | Collaboration Partners | Alnylam | REDEEMABLE CONVERTIBLE PREFERRED STOCK | COMMON STOCK | COMMON STOCKCollaboration Partners | COMMON STOCKAlnylam | ADDITIONAL PAID-IN CAPITAL | ADDITIONAL PAID-IN CAPITALCollaboration Partners | ADDITIONAL PAID-IN CAPITALAlnylam | ACCUMULATED DEFICIT |
Balance at beginning of period at Dec. 31, 2015 | $ 91,022 | $ 2 | $ 287,263 | $ (196,243) | |||||||
Balance at beginning of period (in shares) at Dec. 31, 2015 | 20,594,575 | ||||||||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||||||||
Deemed dividend related to beneficial conversion feature of redeemable convertible preferred stock | 0 | ||||||||||
Exercises of common stock options and sales of common stock under Employee Stock Purchase Plan | 561 | 561 | |||||||||
Exercises of common stock options and sales of common stock under Employee Stock Purchase Plan (in shares) | 152,200 | ||||||||||
Vesting of restricted common stock | 0 | ||||||||||
Vesting of restricted common stock (in shares) | 10,000 | ||||||||||
Settlement of restricted stock for tax withholding | (27) | (27) | |||||||||
Settlement of restricted stock for tax withholding (in shares) | (3,774) | ||||||||||
Stock-based compensation expense | 9,165 | 9,165 | |||||||||
Net loss | (59,513) | (59,513) | |||||||||
Balance at end of period at Dec. 31, 2016 | 41,208 | $ 2 | 296,962 | (255,756) | |||||||
Balance at end of period (in shares) at Dec. 31, 2016 | 20,753,001 | ||||||||||
Balance at beginning of period, temporary equity at Dec. 31, 2015 | $ 0 | ||||||||||
Balance at beginning of period, temporary equity (in shares) at Dec. 31, 2015 | 0 | ||||||||||
Increase (Decrease) in Temporary Equity [Roll Forward] | |||||||||||
Conversion of redeemable convertible preferred stock | 0 | ||||||||||
Balance at end of period, temporary equity at Dec. 31, 2016 | $ 0 | ||||||||||
Balance at end of period, temporary equity (in shares) at Dec. 31, 2016 | 0 | ||||||||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||||||||
Issuance of common stock | 42,779 | $ 1 | 42,778 | ||||||||
Issuance of common stock (in shares) | 6,571,428 | ||||||||||
Beneficial conversion feature, redeemable convertible preferred stock | 6,144 | $ (6,144) | 6,144 | ||||||||
Deemed dividend related to beneficial conversion feature of redeemable convertible preferred stock | (6,144) | 6,144 | (6,144) | ||||||||
Dividends declared, redeemable convertible preferred stock | (9,361) | $ 9,361 | (9,361) | ||||||||
Dividends declared, redeemable convertible preferred stock (in shares) | 55,124 | ||||||||||
Exercises of common stock options and sales of common stock under Employee Stock Purchase Plan | 290 | 290 | |||||||||
Exercises of common stock options and sales of common stock under Employee Stock Purchase Plan (in shares) | 107,523 | ||||||||||
Vesting of restricted common stock | 0 | ||||||||||
Vesting of restricted common stock (in shares) | 10,000 | ||||||||||
Settlement of restricted stock for tax withholding | (11) | (11) | |||||||||
Settlement of restricted stock for tax withholding (in shares) | (3,774) | ||||||||||
Stock-based compensation expense | 7,770 | 7,770 | |||||||||
Net loss | (60,200) | (60,200) | |||||||||
Balance at end of period at Dec. 31, 2017 | 101,086 | $ 5 | 417,037 | (315,956) | |||||||
Balance at end of period (in shares) at Dec. 31, 2017 | 51,644,841 | ||||||||||
Increase (Decrease) in Temporary Equity [Roll Forward] | |||||||||||
Issuance of redeemable convertible preferred stock, net of issuance costs of $750 (in shares) | 700,000 | ||||||||||
Issuance of redeemable convertible preferred stock, net of issuance costs of $750 | 0 | $ 69,250 | |||||||||
Accretion of share issuance costs on redeemable convertible preferred stock | (750) | 750 | (750) | ||||||||
Conversion of redeemable convertible preferred stock | 79,361 | $ (79,361) | $ 2 | 79,359 | |||||||
Conversion of redeemable convertible preferred stock (in shares) | (755,124) | 24,206,663 | |||||||||
Balance at end of period, temporary equity at Dec. 31, 2017 | $ 0 | ||||||||||
Balance at end of period, temporary equity (in shares) at Dec. 31, 2017 | 0 | ||||||||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | |||||||||||
Issuance of common stock | 107,770 | $ 60,412 | $ 10,315 | $ 1 | $ 1 | 107,769 | $ 60,411 | $ 10,315 | |||
Issuance of common stock (in shares) | 8,832,565 | 6,250,019 | 983,208 | ||||||||
Exercise of warrants to purchase common stock | $ 49 | 49 | |||||||||
Exercise of warrants of common stock (in shares) | 329,934 | 45,710 | |||||||||
Deemed dividend related to beneficial conversion feature of redeemable convertible preferred stock | $ 0 | ||||||||||
Exercises of common stock options and sales of common stock under Employee Stock Purchase Plan | 2,061 | 2,061 | |||||||||
Exercises of common stock options and sales of common stock under Employee Stock Purchase Plan (in shares) | 448,173 | ||||||||||
Vesting of restricted common stock | 0 | ||||||||||
Vesting of restricted common stock (in shares) | 10,000 | ||||||||||
Settlement of restricted stock for tax withholding | (35) | (35) | |||||||||
Settlement of restricted stock for tax withholding (in shares) | (3,774) | ||||||||||
Stock-based compensation expense | 7,888 | 7,888 | |||||||||
Net loss | (88,853) | (88,853) | |||||||||
Balance at end of period at Dec. 31, 2018 | 200,693 | $ 7 | $ 605,495 | $ (404,809) | |||||||
Balance at end of period (in shares) at Dec. 31, 2018 | 68,210,742 | ||||||||||
Increase (Decrease) in Temporary Equity [Roll Forward] | |||||||||||
Conversion of redeemable convertible preferred stock | $ 0 | ||||||||||
Balance at end of period, temporary equity at Dec. 31, 2018 | $ 0 | ||||||||||
Balance at end of period, temporary equity (in shares) at Dec. 31, 2018 | 0 |
CONSOLIDATED STATEMENTS OF CH_2
CONSOLIDATED STATEMENTS OF CHANGES IN REDEEMABLE CONVERTIBLE PREFERRED STOCK AND STOCKHOLDERS' EQUITY (Parenthetical) - USD ($) $ in Thousands | Dec. 18, 2017 | Dec. 31, 2018 | Dec. 31, 2017 |
Underwriters’ commissions and offering costs | $ 3,200 | $ 330 | $ 3,221 |
REDEEMABLE CONVERTIBLE PREFERRED STOCK | |||
Issuance costs | $ 750 |
CONSOLIDATED STATEMENTS OF CASH
CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
CASH FLOWS FROM OPERATING ACTIVITIES: | |||
Net loss | $ (88,853) | $ (60,200) | $ (59,513) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Non-cash litigation expense | 10,315 | 0 | 0 |
Stock-based compensation expense | 7,888 | 7,770 | 9,165 |
Depreciation and amortization expense | 774 | 778 | 840 |
Loss on disposal of property and equipment | 12 | 51 | 0 |
Amortization of (premium) discount on investments | (1,126) | (169) | 73 |
Changes in operating assets and liabilities: | |||
Litigation settlement payable | 10,500 | 0 | 0 |
Deferred revenue | 173,916 | 9,270 | 0 |
Prepaid expenses and other assets | 532 | (1,459) | (414) |
Accounts payable | (1,217) | 626 | 1,644 |
Contract receivables | (100,000) | 0 | 0 |
Withholding tax receivable | 1,583 | (1,583) | 0 |
Accrued expenses and other liabilities | 3,974 | (411) | (542) |
Net cash provided by (used in) operating activities | 18,298 | (45,327) | (48,747) |
CASH FLOWS FROM INVESTING ACTIVITIES: | |||
Maturities of held-to-maturity investments | 81,000 | 70,000 | 48,500 |
Purchases of held-to-maturity investments | (283,372) | (89,719) | (35,031) |
Purchases of property and equipment | (359) | (133) | (449) |
Net cash (used in) provided by investing activities | (202,731) | (19,852) | 13,020 |
CASH FLOWS FROM FINANCING ACTIVITIES: | |||
Proceeds from issuance of common stock, net of underwriters’ commissions | 108,099 | 43,225 | 0 |
Payments of common stock offering costs | (703) | (23) | 0 |
Proceeds from issuance of redeemable convertible preferred stock | 0 | 70,000 | 0 |
Redeemable convertible preferred stock issuance costs | 0 | (750) | 0 |
Proceeds from issuance of common stock to collaboration partners | 60,412 | 0 | 0 |
Proceeds from exercises of common stock warrants, stock options and issuances under Employee Stock Purchase Plan | 2,110 | 290 | 561 |
Settlement of restricted stock for tax withholding | (35) | (11) | (27) |
Net cash provided by financing activities | 169,883 | 112,731 | 534 |
NET (DECREASE) INCREASE IN CASH, CASH EQUIVALENTS, AND RESTRICTED CASH EQUIVALENTS | (14,550) | 47,552 | (35,193) |
CASH, CASH EQUIVALENTS, AND RESTRICTED CASH EQUIVALENTS – Beginning of year | 69,533 | 21,981 | 57,174 |
CASH, CASH EQUIVALENTS AND RESTRICTED CASH EQUIVALENTS – End of year | 54,983 | 69,533 | 21,981 |
NONCASH INVESTING ACTIVITIES: | |||
Property and equipment purchases included in accounts payable and accrued expenses | 1,648 | 15 | 53 |
NONCASH FINANCING ACTIVITIES: | |||
Conversion of redeemable convertible preferred stock into common stock | 0 | 79,361 | 0 |
Dividends on redeemable convertible preferred stock | 0 | 10,111 | 0 |
Deemed dividend related to beneficial conversion feature of redeemable convertible preferred stock | 0 | 6,144 | 0 |
Deemed dividend on conversion of redeemable convertible preferred stock | 0 | 3,837 | 0 |
Common stock offering costs included in accounts payable or accrued expenses | 50 | 423 | 0 |
Cash, Cash Equivalents, Restricted Cash and Restricted Cash Equivalents [Abstract] | |||
Total cash, cash equivalents, and restricted cash equivalents shown in the consolidated statements of cash flows | $ 69,533 | $ 21,981 | $ 57,174 |
DESCRIPTION OF BUSINESS AND BAS
DESCRIPTION OF BUSINESS AND BASIS OF PRESENTATION | 12 Months Ended |
Dec. 31, 2018 | |
Accounting Policies [Abstract] | |
DESCRIPTION OF BUSINESS AND BASIS OF PRESENTATION | DESCRIPTION OF BUSINESS AND BASIS OF PRESENTATION Business Dicerna ™ Pharmaceuticals, Inc. (“Dicerna” or the “Company”), a Delaware corporation founded in 2006 and headquartered in Cambridge, Massachusetts, is a biopharmaceutical company focused on the discovery and development of innovative subcutaneously delivered ribonucleic acid (“RNA”) interference (“RNAi”)-based pharmaceuticals using its GalXC ™ RNAi platform for the treatment of diseases involving the liver, including rare diseases, viral infectious diseases, chronic liver diseases, and cardiovascular diseases. Within these therapeutic areas, the Company believes its GalXC RNAi platform will allow the Company to build a broad pipeline of therapeutics with attractive pharmaceutical properties, including a subcutaneous route of administration, infrequent dosing (e.g., dosing that is monthly or quarterly, and potentially even less frequent), high therapeutic index, and specificity to a single target gene. Basis of presentation The accompanying consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America (“GAAP”) and include the accounts of Dicerna Pharmaceuticals, Inc. and its wholly-owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. Reclassifications Effective January 1, 2018, the Company changed the presentation of the income from government grants from the caption “Grant revenue” to an offset to research and development expenses. Also, effective April 1, 2018, the Company changed the presentation of certain litigation-related expenses associated with the litigation with Alnylam Pharmaceuticals, Inc. (“Alnylam”) from the caption “General and administrative” expense to “Litigation expense.” The changes associated with changes in presentation were applied retrospectively through the recast of affected prior period amounts in the consolidated statements of operations. The primary effects of such changes were: • the reclassification of grant income from revenue to the presentation as an offset to research and development expenses of $1.1 million and $0.3 million for the years ended December 31, 2017 and 2016, respectively; and • the reclassification of certain litigation-related expenses historically included in general and administrative expense to litigation expense in the consolidated statements of operations of $9.0 million and $2.9 million for the years ended December 31, 2017 and 2016, respectively. Significant judgments and estimates The preparation of consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, and the disclosure of contingent assets and liabilities at the date of the consolidated financial statements, as well as the revenues and expenses incurred during the reporting periods. On an ongoing basis, the Company evaluates its judgments and estimates, including those related to revenue recognition and accrued expenses. The Company bases its estimates on historical experience and on various other factors that the Company believes are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not apparent from other sources. Changes in estimates are reflected in reported results for the period in which they become known. Actual results could differ materially from those estimates. Liquidity The Company had cash, cash equivalents, and held-to-maturity investments of $302.6 million as of December 31, 2018 . The Company believes that its current cash, cash equivalents, and held-to-maturity investments as of December 31, 2018 will be sufficient to fund the execution of its current clinical and operating plan beyond 2020. This estimate assumes no new funding from additional collaboration agreements or from external financing events and no significant unanticipated changes in costs and expenses. In early 2019, the Company received $5.0 million from BI for the Option Payment and $100.0 million for the upfront cash payment from the Company’s recent collaboration with Eli Lilly and Company (“Lilly”), and also paid $10.5 million to Alnylam for the Confidential Settlement Agreement and General Release (“Settlement Agreement”). |
SUMMARY OF SIGNIFICANT ACCOUNTI
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | 12 Months Ended |
Dec. 31, 2018 | |
Accounting Policies [Abstract] | |
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES Cash and cash equivalents Cash and cash equivalents includes all highly liquid investments, including money market funds, maturing within 90 days from the date of purchase. Restricted cash equivalents Restricted cash equivalents includes the balance of funds held in a money market collateral account that is restricted to secure a letter of credit for the Company’s operating lease for office and laboratory space at 87 Cambridgepark Drive in Cambridge, Massachusetts. The letter of credit is required to be maintained throughout the term of the Company’s lease, which expires on November 30, 2020 . Concentrations of credit risk and significant customers Financial instruments that subject the Company to significant concentrations of credit risk consist of cash, cash equivalents, restricted cash equivalents, held-to-maturity investments, contract receivables, and the withholding tax receivable (see Note 8 – Collaborative Research and License Agreements). All of the Company’s cash, cash equivalents, restricted cash equivalents, and held-to-maturity investments are invested in money market funds or United States (“U.S.”) treasury securities that management believes to be of high credit quality. The Company’s revenues for the year ended December 31, 2018 are a result of the Company’s collaboration agreements with Boehringer Ingelheim (“BI”) and Alexion Pharmaceuticals, Inc. (“Alexion”). BI represented substantially all of the Company’s revenue from collaborative arrangements for the years ended December 31, 2018 and 2017 . All revenues recognized by the Company to date were earned in the U.S. At December 31, 2018 , the balance of the Company’s contract receivables was solely related to the non-refundable, non-creditable upfront payment due to the Company in connection with a collaboration agreement entered into with Lilly (see Note 8 – Collaborative Research and License Agreements ). The Company did no t have any contract receivables at December 31, 2017 . The Company does not currently own or operate any manufacturing facilities for the production of preclinical, clinical, or commercial quantities of any of its product candidates. For each product candidate, the Company currently contracts with manufacturers, and expects to continue to do so to meet the preclinical and clinical requirements of its product candidates. For the year ended December 31, 2018 , the Company had one contract manufacturing relationship which accounted for approximately 10.9% of the Company’s total purchases. Property and equipment Property and equipment are stated at cost. Major betterments are capitalized whereas expenditures for maintenance and repairs which do not improve or extend the life of the respective assets are charged to operations as incurred. Depreciation is provided using the straight-line method over the estimated useful lives, as shown below: ASSET CATEGORY ESTIMATED Laboratory equipment 5 years Office and computer equipment 3 - 5 years Furniture and fixtures 5 years Leasehold improvements 5 years or the remaining Construction in process is stated at cost, which includes the cost of construction and other direct costs attributable to the construction. No provision for depreciation and amortization expense is recorded related to construction in process until the relevant assets are completed and put into use. At December 31, 2018 , the balance of construction in process includes costs associated with laboratory equipment under installation and the construction of certain leasehold improvements. Impairment of long-lived assets Long-lived assets are reviewed for impairment whenever events or changes in circumstances indicate that the carrying amount of the asset may not be recoverable. When such events occur, the Company compares the carrying amounts of the assets to their undiscounted expected future cash flows. If this comparison indicates that there is an impairment, the amount of the impairment is calculated as the difference between the carrying value and fair value of the related asset. During the years ended December 31, 2018 , 2017 , and 2016 , no impairments were recorded. Segment and geographic information Operating segments are defined as components (business activity from which it earns revenue and incurs expenses) of an enterprise about which discrete financial information is available and regularly reviewed by the chief operating decision maker in deciding how to allocate resources and in assessing performance. The Company, through its Chief Executive Officer in his role as chief operating decision maker, views its operations and manages its business as one operating segment. All long-lived assets of the Company are located in the United States. Research and development costs Research and development costs consist of expenses incurred in performing research and development activities, including compensation and benefits for full-time research and development employees, an allocation of facility expenses, overhead expenses and other outside expenses. Research and development costs are expensed as incurred. Research and development costs that are paid in advance of performance are deferred as a prepaid expense and amortized over the service period as the services are provided. The Company records grants from governmental and non-profit agencies as a reduction in research and development expense. Grants are recognized when there is reasonable assurance that the Company will comply with the conditions attached to the grant arrangement and the grant will be received. Grant payments received related to research and development costs incurred prior to the approval of the qualifying program are recognized immediately upon approval of the program by the grantor. Revenue recognition The Company generates revenue from research collaboration and license agreements with customers. Goods and services in the agreements may include the grant of licenses for the use of the Company’s technology, the provision of services associated with the research and development of product candidates, manufacturing services, and participation on joint steering committees. Such agreements may provide for consideration to the Company in the form of upfront payments; funding or reimbursement of research and development services; reimbursement of certain costs; option exercise payments; payments due upon the achievement of preclinical, clinical, regulatory, and sales-based milestones; and royalty payments on licensed products. On January 1, 2018, the Company adopted the new revenue recognition standard, discussed below under the heading “Recent accounting pronouncements,” which amended revenue recognition principles and provides a single, comprehensive set of criteria for revenue recognition. The new revenue standard applies to all contracts with customers except for contracts that are within the scope of other standards. The new guidance provides a five-step framework through which revenue is recognized when control of promised goods or services is transferred to a customer at an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. To determine revenue recognition for arrangements that the Company concludes are within the scope of the new revenue recognition standard, management performs the following five steps: (i) identifies the contract(s) with a customer; (ii) identifies the performance obligations in the contract; (iii) determines the transaction price, including whether there are any constraints on variable consideration; (iv) allocates the transaction price to the performance obligations; and (v) recognizes revenue when (or as) the Company satisfies a performance obligation. At contract inception, once a contract is determined to be within the scope of the new revenue standard, Dicerna assesses whether individual goods or services promised within each contract are distinct and, therefore, represent a separate performance obligation. Goods and services that are determined not to be distinct are combined with other promised goods and services until a distinct bundle is identified. Dicerna allocates the transaction price (the amount of consideration to which the Company expects to be entitled in exchange for the promised goods or services) to each performance obligation and recognizes the associated revenue when (or as) each performance obligation is satisfied. The Company’s estimate of the transaction price for each contract includes all variable consideration to which Dicerna expects to be entitled at each measuring period. When two or more contracts are entered into with the same customer at or near the same time, the Company evaluates the contracts to determine whether the contracts should be accounted for as a single arrangement. Contracts are combined and accounted for as a single arrangement if one or more of the following criteria are met: (i) the contracts are negotiated as a package with a single commercial objective; (ii) the amount of consideration to be paid in one contract depends on the price or performance of the other contract; or (iii) the goods or services promised in the contracts (or some goods or services promised in each of the contracts) are a single performance obligation. The evaluation of whether promised goods or services represent distinct performance obligations is subjective and requires the Company to make judgments about the promised goods and services and whether such goods and services are separable from the other aspects of the contract(s). The transaction price is allocated among the performance obligations on a relative standalone selling price basis, and the applicable revenue recognition criteria are applied to each of the separate performance obligations. The Company may estimate the standalone selling price using a residual method when the selling price is highly variable because a representative standalone selling price is not discernible from past transactions or other observable evidence, or when the selling price is uncertain. Determining the standalone selling price for performance obligations requires significant judgment. When an observable price of a promised good or service is not readily available, the Company considers relevant assumptions to estimate the standalone selling price, including, as applicable, market conditions, development timelines, probabilities of technical and regulatory success, reimbursement rates for personnel costs, forecasted revenues, potential limitations to the selling price of the product, and discount rates. The Company applies judgment in determining whether a combined performance obligation is satisfied at a point in time or over time, and, if over time, concluding upon the appropriate method of measuring progress to be applied for purposes of recognizing revenue. The Company evaluates the measure of progress each reporting period and, as estimates related to the measure of progress change, related revenue recognition is adjusted accordingly. Changes in the Company’s estimated measure of progress are accounted for prospectively as a change in accounting estimate. The Company receives payments from its licensees as established in each contract. Upfront payments and fees are recorded as deferred revenue upon receipt or when due and most often require deferral of revenue recognition to a future period until the Company performs its obligations under the underlying arrangements. Where applicable, amounts are recorded as contracts receivable when the Company’s right to consideration is unconditional. Licenses of intellectual property: If a license granted to a customer to use the Company’s intellectual property is determined to be distinct from the other performance obligations identified in the arrangement, the Company recognizes revenue from consideration allocated to the license when the license is transferred to the licensee and the licensee is able to use and benefit from the license. For licenses that are bundled with other promises, the Company applies judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, to conclude upon the appropriate method of measuring progress for purposes of recognizing revenue related to consideration allocated to the performance obligation. Research and development services: Arrangements that include a promise for the Company to provide research or development services are assessed to determine whether the services are capable of being distinct, are not highly interdependent or do not significantly modify one another, and if so, the services are accounted for as a separate performance obligation as the services are provided to the customer. Otherwise, when research or development services are determined not to be capable of being distinct, such services are added to the performance obligation that includes the underlying license. For research and development services that are bundled with other promises, the Company applies judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, to conclude upon the appropriate method of measuring progress for purposes of recognizing revenue related to consideration allocated to the performance obligation. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition. Options: Customer options, such as options granted to allow a licensee to choose to research and develop additional or reserve product candidates against target genes to be identified in the future, or options that allow a customer to designate a target as a lead product, are evaluated at contract inception in order to determine whether those options provide a material right (i.e., an optional good or service offered for free or at a discount) to the customer. If the customer options represent a material right, the material right is treated as a separate performance obligation at the outset of the arrangement. The Company allocates the transaction price to material rights based on the standalone selling price, and revenue is recognized when or as the future goods or services are transferred or when the option expires. Customer options that are not material rights do not give rise to a separate performance obligation, and as such, the additional consideration that would result from a customer exercising an option in the future is not included in the transaction price for the current contract. Instead, the option is deemed a marketing offer, and additional option fee payments are recognized or begin being recognized as revenue when the licensee exercises the option. The exercise of an option that does not represent a material right is treated as a separate contract for accounting purposes. Milestone payments: At the inception of each contract with a customer that includes development or regulatory milestone payments, the Company evaluates whether the milestones are considered probable of being reached and estimates the amount to be included in the transaction price using the most likely amount method. If the Company concludes it is probable that a significant revenue reversal would not occur, the associated milestone payment is included in the transaction price. Milestone payments that are not within the control of the Company or the licensee, such as regulatory approvals, are generally not considered probable of being achieved until those approvals are received. The transaction price is then allocated to each performance obligation on a relative stand-alone selling price basis, for which the Company recognizes revenue as or when the performance obligations under the contract are satisfied. At the end of each subsequent reporting period, the Company re-evaluates the probability of achievement of all milestones and any related constraints, and, if necessary, adjusts the estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis and are recorded as revenue and through earnings in the period of adjustment. Royalties: For arrangements that include sales-based royalties, including milestone payments based on the level of sales, and when the license is deemed to be the predominant item to which the royalties relate, the Company recognizes revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). Contract costs: The Company recognizes as an asset the incremental costs of obtaining a contract with a customer if the costs are expected to be recovered. The Company has elected a practical expedient wherein it recognizes the incremental costs of obtaining a contract as an expense when incurred if the amortization period of the asset that it otherwise would have recognized is one year or less. To date, the Company has not incurred any incremental costs of obtaining a contract with a customer. Contract modifications: Contract modifications, defined as changes in the scope or price (or both) of a contract that are approved by the parties to the contract, such as a contract amendment, exist when the parties to a contract approve a modification that either creates new or changes existing enforceable rights and obligations of the parties to the contract. Depending on facts and circumstances, the Company accounts for a contract modification as one of the following: (i) a separate contract; (ii) a termination of the existing contract and a creation of a new contract; or (iii) a combination of the preceding treatments. A contract modification is accounted for as a separate contract if the scope of the contract increases because of the addition of promised goods or services that are distinct and the price of the contract increases by an amount of consideration that reflects the Company’s standalone selling prices of the additional promised goods or services. When a contract modification is not considered a separate contract and the remaining goods or services are distinct from the goods or services transferred on or before the date of the contract modification, the Company accounts for the contract modification as a termination of the existing contract and a creation of a new contract. When a contract modification is not considered a separate contract and the remaining goods or services are not distinct, the Company accounts for the contract modification as an add-on to the existing contract and as an adjustment to revenue on a cumulative catch-up basis. The Company receives payments from its licensees as established in each contract. Upfront payments and fees are recorded as deferred revenue upon receipt or when due and may require deferral of revenue recognition to a future period until the Company performs its obligations under these arrangements. Where applicable, amounts are recorded as contracts receivable when the Company’s right to consideration is unconditional. The Company does not assess whether a contract with a customer has a significant financing component if the expectation at contract inception is such that the period between payment by the licensees and the transfer of the promised goods or services to the licensees will be one year or less. Stock-based compensation The Company’s stock-based compensation cost is measured at the grant date of the stock-based award based on the fair value of the award and is recognized as expense over the requisite service period, which generally represents the vesting period, and includes an estimate of the awards that will be forfeited. The Company uses the Black-Scholes valuation model for estimating the fair value of stock options. The fair value of stock option awards is affected by the valuation assumptions, including the expected volatility based on comparable market participants, expected term of the option, risk-free interest rate and expected dividends. Income taxes The Company records deferred tax assets and liabilities for the expected future tax consequences of temporary differences between the Company’s financial statement carrying amounts and the tax basis of assets and liabilities using enacted tax rates expected to be in effect in the years in which the differences are expected to reverse. A valuation allowance is provided to reduce the net deferred tax assets to the amount that will more likely than not be realized. The Company also assesses the probability that the positions taken or expected to be taken in its income tax returns will be sustained by taxing authorities. A “more likely than not” (more than 50 percent ) recognition threshold must be met before a tax benefit can be recognized. Tax positions that are more likely than not to be sustained are reflected in the Company’s consolidated financial statements. Tax positions are measured as the largest amount of tax benefit that is greater than 50 percent likely of being realized upon settlement with a taxing authority that has full knowledge of all relevant information. The difference between the benefit recognized for a position and the tax benefit claimed on a tax return is referred to as an unrecognized tax benefit. Potential interest and penalties associated with such uncertain tax positions are recorded as a component of income tax expense. Net loss per common share attributable to common stockholders The Company computes basic net loss per common share by dividing net loss attributable to common stockholders by the weighted‑average number of common shares outstanding. In periods of net income, the Company’s accounting policy includes allocating a proportional share of net income to participating securities, as determined by dividing total weighted‑average participating securities by the sum of the total weighted‑average common shares and participating securities (the “two-class method”). The Company’s nonvested restricted shares participated in any dividends declared by the Company and were therefore considered to be participating securities. Participating securities have the effect of diluting both basic and diluted earnings per share during periods of income. During periods when the Company incurred a net loss, the Company did not allocate a loss to participating securities because they had no contractual obligation to share in the losses of the Company. The Company computes diluted net loss per common share after giving consideration to the dilutive effect of stock options, warrants, nonvested restricted stock, and redeemable convertible preferred shares that are outstanding during the period, except where such non-participating securities would be anti-dilutive. The outstanding securities presented below were excluded from the calculation of net loss per share attributable to common stockholders because such securities would have been anti-dilutive due to the Company’s net loss per share attributable to common stockholders during the periods ending on the dates presented. DECEMBER 31, 2018 2017 2016 Options to purchase common stock 7,787,690 6,124,096 5,099,449 Warrants to purchase common stock 2,198 87,901 87,901 Nonvested restricted common stock — 10,000 20,000 Total 7,789,888 6,221,997 5,207,350 Comprehensive loss Comprehensive loss is defined as the change in stockholders’ equity of a business enterprise during a period from transactions and other events and circumstances from non-owner sources. The Company has no comprehensive loss items other than net loss. Recent accounting pronouncements The following table provides a description of the recent accounting pronouncements that may have a material effect on the Company’s consolidated financial statements: Standard Description Effective Date Effect on the Consolidated Accounting Standards Adopted During the Year Ended December 31, 2018 ASU 2014-09, Revenue from Contracts with Customers (Topic 606) and related amendments (“ASC 606”) This ASU amends the guidance for accounting for revenue from contracts with customers, superseding the revenue recognition requirements in ASC 605, Revenue Recognition . ASC 606 was effective for annual reporting periods beginning after December 15, 2017. Under ASC 606, two adoption methods were allowed: retrospectively to all prior reporting periods presented, with certain practical expedients permitted, or retrospectively with the cumulative effect of initially adopting ASC 606 recognized at the date of initial application. January 1, 2018 Effective January 1, 2018, the Company adopted the requirements of ASC 606 using the full retrospective method, which required the Company to recast the prior reporting periods presented. All financial statements and disclosures have been recast to comply with ASC 606. See “Change in accounting principle” below for a summary of the amounts by which each financial statement line item was affected by the adoption of ASC 606. – Collaborative Research and License Agreements ). ASU No. 2016-18, Statement of Cash Flows (Topic 230): Restricted Cash (“ASU 2016-18”) In November 2016, the FASB issued ASU No. 2016-18, Statement of Cash Flows (Topic 230): Restricted Cash (“ASU 2016-18”), a consensus of the FASB’s EITF. ASU 2016-18 requires that the statement of cash flows explain the change during the period in the total of cash and cash equivalents, including amounts generally described as restricted cash or restricted cash equivalents. Entities are required to reconcile such total to amounts on the balance sheet and disclose the nature of the restrictions. By requiring that the statement of cash flows explain the change during the period in the total of cash, cash equivalents and restricted cash, the new guidance eliminates current diversity in practice. January 1, 2018 The Company adopted ASU 2016-18 on January 1, 2018 and applied this new guidance retrospectively to all periods presented. Consequently, transfers between restricted and unrestricted cash equivalents accounts are no longer reported as a cash flow in the Company’s consolidated statement of cash flows. As a result of the adoption of this standard, the Company includes its restricted cash equivalents balance in the cash and cash equivalents reconciliation of operating, investing and financing activities. The retrospective adoption resulted in the inclusion of restricted cash equivalents of $0.7 million, and $1.1 million in the consolidated statements of cash flows as of December 31, 2017 and 2016, respectively. Standard Description Effective Date for Company Effect on the Consolidated Financial Statements Recently Issued Accounting Standards Not Yet Adopted ASU 2016-02, Leases (Topic 842) This ASU supersedes existing accounting standards for lease accounting, including requiring lessees to recognize most leases on their balance sheets and making targeted changes to lessor accounting. ASU 2016-02 will be effective for the Company beginning in the first quarter of 2019. ASU 2016-02 requires a modified retrospective transition approach for all leases existing at, or entered into after, the date of initial application, with an option to use certain transition relief. In July 2018, the FASB issued ASU 2018-11, Leases (Topic 842): Targeted Improvements (“ASU 2018-11”), which allows entities to initially apply the new lease guidance at the adoption date and recognize a cumulative-effect adjustment to the opening balance of retained earnings in the period of adoption. January 1, 2019 Management expects that the adoption of ASU 2016-02 will result in the recognition of a right of use asset and related liability associated with the Company’s non-cancelable operating lease arrangements for office and laboratory spaces (see Note 14 – Commitments and Contingencies and Note 17 - Subsequent Events ). The Company is in the process of determining whether it will utilize the optional transition method presented in ASU 2018-11. Change in accounting principle In May 2014, the Financial Accounting Standards Board ("FASB") issued ASC 606. Under the standard, revenue is recognized when a customer obtains control of promised goods or services in an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. Effective January 1, 2018, the Company adopted ASC 606 using the full retrospective method, which required the Company to recast the prior reporting periods presented. The Company has recast its consolidated financial statements from amounts previously reported due to the adoption of ASC 606. Select Consolidated Statement of Operations line items, which reflect the impact of the adoption of ASC 606, are as follows: YEAR ENDED DECEMBER 31, 2017 AS REPORTED ADJUSTMENTS AS ADJUSTED Revenue from collaborative arrangements $ 1,182 $ (152 ) $ 1,030 Loss from operations $ (60,587 ) $ (152 ) $ (60,739 ) Net loss $ (60,048 ) $ (152 ) $ (60,200 ) Net loss attributable to common stockholders $ (80,140 ) $ (152 ) $ (80,292 ) The adoption of ASC 606 did not have an impact on net loss per share attributable to common stockholders for any period presented. Select Consolidated Balance Sheet line items, which reflect the adoption of ASC 606, are as follows: DECEMBER 31, 2017 AS REPORTED ADJUSTMENTS AS ADJUSTED Prepaid expenses and other current assets $ 3,297 $ 118 $ 3,415 Current portion of deferred revenue $ 6,000 $ 180 $ 6,180 Deferred revenue, net of current portion $ 3,000 $ 90 $ 3,090 Accumulated deficit $ (315,804 ) $ (152 ) $ (315,956 ) The adoption of ASC 606 did not have an impact on net cash used in operating, investing, or financing activities in the Company’s Consolidated Statements of Cash Flows. As the Company did no t have any revenue from collaborative arrangements during the year ended December 31, 2016, the adoption of ASC 606 did not have an impact on the beginning balance of accumulated deficit for the earliest period presented in the Consolidated Statements of Changes in Redeemable Convertible Preferred Stock and Stockholders’ Equity. |
HELD-TO-MATURITY INVESTMENTS
HELD-TO-MATURITY INVESTMENTS | 12 Months Ended |
Dec. 31, 2018 | |
Investments, Debt and Equity Securities [Abstract] | |
HELD-TO-MATURITY INVESTMENTS | HELD-TO-MATURITY INVESTMENTS The Company invests its excess cash balances in short-term and long-term fixed-income investments. The Company determines the appropriate classification of investments at the time of purchase and re-evaluates such designation as of each balance sheet date. Debt securities carried at amortized cost are classified as held-to-maturity when the Company has the positive intent and ability to hold the securities to maturity. The Company’s investment policy mandates that, at the time of purchase, the maturity of each investment within its portfolio shall not exceed 24 months . In addition, the weighted‑average maturity of the investment portfolio must not exceed 12 months . The following tables provide information relating to the Company’s held-to-maturity investments: DECEMBER 31, 2018 DESCRIPTION AMORTIZED COST GROSS UNREALIZED GAINS GROSS LOSSES FAIR VALUE U.S. Treasury securities maturing in one year or less $ 248,387 $ — $ (43 ) $ 248,344 DECEMBER 31, 2017 DESCRIPTION AMORTIZED COST GROSS UNREALIZED GAINS GROSS LOSSES FAIR VALUE U.S. Treasury securities maturing in one year or less $ 44,889 $ — $ (30 ) $ 44,859 |
FAIR VALUE MEASUREMENTS
FAIR VALUE MEASUREMENTS | 12 Months Ended |
Dec. 31, 2018 | |
Fair Value Disclosures [Abstract] | |
FAIR VALUE MEASUREMENTS | FAIR VALUE MEASUREMENTS Fair value is an exit price, representing the amount that would be received from the sale of an asset or paid to transfer a liability in an orderly transaction between market participants. As such, fair value is a market-based measurement that should be determined based on assumptions that market participants would use in pricing an asset or liability. Valuation techniques used to measure fair value are performed in a manner to maximize the use of observable inputs and minimize the use of unobservable inputs. As a basis for considering such assumption the accounting literature establishes a three-tier value hierarchy which prioritizes the inputs used in measuring fair value as follows: • Level 1 – observable inputs, such as quoted prices in active markets; • Level 2 – inputs other than the quoted prices in active markets that are observable either directly or indirectly; and • Level 3 – unobservable inputs for which there is little or no market data, which requires the Company to develop its own assumptions. A summary of the Company’s assets that are measured or disclosed at fair value on a recurring basis is presented below: DECEMBER 31, 2018 DESCRIPTION TOTAL FAIR VALUE LEVEL 1 LEVEL 2 LEVEL 3 Cash equivalents Money market funds $ 44,886 $ 44,886 $ — $ — Held-to-maturity investment U.S. Treasury securities 248,344 — 248,344 — Restricted cash equivalents Money market funds 744 — 744 — Total $ 293,974 $ 44,886 $ 249,088 $ — DECEMBER 31, 2017 DESCRIPTION TOTAL FAIR VALUE LEVEL 1 LEVEL 2 LEVEL 3 Cash equivalents Money market funds $ 51,441 $ 51,441 $ — $ — Held-to-maturity investments U.S. Treasury securities 44,859 — 44,859 — Restricted cash equivalents Money market funds 744 — 744 — Total $ 97,044 $ 51,441 $ 45,603 $ — The Company’s cash equivalents, which are in money market funds, are classified within Level 1 of the fair value hierarchy because they are valued using quoted prices in active markets as of December 31, 2018 and 2017 . The Company’s held-to-maturity investments and restricted cash equivalents bore interest at the prevailing market rates for instruments with similar characteristics and therefore approximated fair value. These financial instruments were classified within Level 2 of the fair value hierarchy, because the inputs to the fair value measurement are valued using observable inputs as of December 31, 2018 and 2017 . As of December 31, 2018 and 2017 , the Company’s contract receivables, accounts payable, and accrued expenses approximated their estimated fair values because of the short-term nature of these financial instruments. As of December 31, 2017, the carrying amount of the withholding tax receivable also approximated its estimated fair value due to the short-term nature of the instrument. As of December 31, 2018 , the Company had a remaining cash obligation of $10.5 million payable to Alnylam (see Note 15 ). Upon receipt of certain upfront cash payment owed to the Company resulting from signing the Lilly Collaboration Agreement in October 2018, the Company anticipates that the cash obligation will be payable in the first quarter of 2019 and has therefore adjusted the liability equal to the estimated present value of the obligation of $10.5 million and included the obligation in current liabilities at December 31, 2018 . As the present value of the litigation settlement payable at December 31, 2018 was determined using market rates based on the nature of the obligation and the Company’s creditworthiness, the carrying value approximates the fair value. There was no liability recorded related to the settlement as of December 31, 2017 . The Company’s policy is to recognize transfers between levels of the fair value hierarchy, if any, at the end of the reporting period; however, there have been no such transfers during any of the periods presented. |
PREPAID EXPENSES AND OTHER CURR
PREPAID EXPENSES AND OTHER CURRENT ASSETS | 12 Months Ended |
Dec. 31, 2018 | |
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract] | |
PREPAID EXPENSES AND OTHER CURRENT ASSETS | PREPAID EXPENSES AND OTHER CURRENT ASSETS Prepaid expenses and other current assets consist of the following: DECEMBER 31, 2018 2017 Prepaid clinical, contract research, and manufacturing costs $ 1,419 $ 1,931 Interest receivable and other current assets 815 391 Prepaid insurance 341 318 Prepaid rent 245 239 Other 68 536 Prepaid expenses and other current assets $ 2,888 $ 3,415 |
PROPERTY AND EQUIPMENT, NET
PROPERTY AND EQUIPMENT, NET | 12 Months Ended |
Dec. 31, 2018 | |
Property, Plant and Equipment [Abstract] | |
PROPERTY AND EQUIPMENT, NET | PROPERTY AND EQUIPMENT, NET Property and equipment, net, consists of the following : DECEMBER 31, 2018 2017 Laboratory equipment $ 4,607 $ 4,410 Office and computer equipment 1,021 900 Furniture and fixtures 479 479 Leasehold improvements 257 257 Construction in process 1,661 — Property and equipment, at cost 8,025 6,046 Less accumulated depreciation and amortization (5,307 ) (4,534 ) Property and equipment, net $ 2,718 $ 1,512 Depreciation and amortization expense was $0.8 million for each of the years ended December 31, 2018 , 2017 , and 2016 . |
ACCRUED EXPENSES AND OTHER CURR
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES | 12 Months Ended |
Dec. 31, 2018 | |
Payables and Accruals [Abstract] | |
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES | ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES Accrued expenses and other current liabilities consist of the following: DECEMBER 31, 2018 2017 Accrued clinical, contract research, and manufacturing costs $ 3,960 $ 1,860 Accrued compensation and related benefits 3,684 1,987 Accrued professional fees 1,693 1,488 Accrued other expenses 312 391 Accrued expenses and other current liabilities $ 9,649 $ 5,726 |
COLLABORATIVE RESEARCH AND LICE
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS | 12 Months Ended |
Dec. 31, 2018 | |
Revenue from Contract with Customer [Abstract] | |
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS | COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS Lilly collaboration and share purchase agreements On October 25, 2018 , the Company entered into a Collaboration and License Agreement (the “Lilly Collaboration Agreement”) with Lilly for the discovery, development, and commercialization of potential new medicines in the areas of cardiometabolic disease, neurodegeneration, and pain. Under the terms of the Lilly Collaboration Agreement, the Company and Lilly will seek to use the Company’s proprietary GalXC RNAi technology platform to progress new drug targets toward clinical development and commercialization. In addition, the Company and Lilly will collaborate to extend the GalXC RNAi platform technology to non-liver (i.e., non-hepatocyte) tissues, including neural tissues. The Lilly Collaboration Agreement provides that the Company will work exclusively with Lilly in the neurodegeneration and pain fields, with the exception of mutually agreed upon orphan indications. Additionally, the Company will work exclusively with Lilly on select targets in the cardiometabolic field. Under the Lilly Collaboration Agreement, the Company will provide Lilly with exclusive and non-exclusive licenses to support the companies’ activities and to enable Lilly to commercialize products derived from or containing compounds developed pursuant to such agreement. The Lilly Collaboration Agreement provides for three initially named hepatocyte targets, and the Company and Lilly have agreed to develop an initial research program with the goal of researching and developing multiple lead candidates directed to each of these initial targets. The Lilly Collaboration Agreement contemplates in excess of 10 targets. Under the terms of the Lilly Collaboration Agreement, Lilly agreed to pay the Company a non-refundable, non-creditable upfront payment of $100.0 million . The Company is also eligible to receive up to $350.0 million , per target, in development and commercialization milestones, in addition to a $5.0 million payment, which will become due for each of the non-hepatocyte targets when a product candidate achieves proof of principle in an animal model. In addition, the Company is eligible to earn mid-single to low-double digit royalties on product sales on a country-by-country and product-by-product basis until the later of expiration of patent rights in a country, the expiration of data or regulatory exclusivity in such country, or 10 years after the first product sale in such country, subject to certain royalty step-down provisions set forth in the agreement. Simultaneously with the entry into the Lilly Collaboration Agreement, the Company and Lilly entered into a Share Purchase Agreement (the “Lilly Share Issuance Agreement”), pursuant to which Lilly purchased 5,414,185 shares of the Company’s common stock at $18.47 per share, for an aggregate purchase price of $100.0 million . Management concluded that the Lilly Share Issuance Agreement is to be combined with the Lilly Collaboration Agreement (together, the “Combined Agreements”) for accounting purposes. Of the total $200.0 million upfront compensation, the Company applied equity accounting guidance to measure the $51.3 million recorded in equity upon the issuance of the shares, and $148.7 million was identified as the transaction price allocated to the revenue arrangement. The Combined Agreements were subject to customary closing conditions and clearances, including clearance under the Hart-Scott-Rodino Antitrust Improvements Act, and closed in December 2018. The Company concluded that Lilly is a customer in this arrangement, and as such, the element of the arrangement unrelated to the issuance of the shares falls within the scope of the revenue recognition guidance. The Company identified contract promises under the Combined Agreements for licenses of intellectual property and know-how rights, associated research and development services for targets and for a new platform, and participation on a joint steering committee. The Company d etermined that the performance obligations were not separately identifiable and were not distinct or distinct within the context of the contract due to the specialized nature of the services to be provided by Dicerna, specifically with respect to the Company’s therapeutic expertise related to RNAi and the Company’s GalXC conjugates, and the interdependent relationship between the performance obligations. As such, the Company concluded that there is a single identified combined performance obligation. The Company used the most likely amount method to estimate variable consideration and estimated that the most likely amount for each potential development milestone payment under this agreement, which is considered variable consideration, was zero , as the achievement of those milestones is uncertain and highly susceptible to factors outside of the Company’s control. Accordingly, all such milestones were excluded from the transaction price. Management will re-evaluate the transaction price at the end of each reporting period and as uncertain events are resolved or other changes in circumstances occur and adjust the transaction price as necessary. Sales-based royalties, including milestone payments based on the level of sales, were also excluded from the transaction price, as the license is deemed to be the predominant item to which the royalties relate. The Company will recognize such revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). Revenue associated with the performance obligation will be recognized as services are provided using a cost-to-cost measure of progress method. The transfer of control occurs over time and, in management’s judgment, this input method is the best measure of progress towards satisfying the performance obligation and reflects a faithful depiction of the transfer of goods and services. No revenue was recognized under the Lilly Collaboration Agreement during the year ended December 31, 2018 . The aggregate amount of the consideration received and the amount billed under the arrangement that were allocated to the revenue element of the arrangement as of December 31, 2018 relates to the Company’s wholly unsatisfied performance obligation. This amount is recorded as a contract liability presented in deferred revenue at December 31, 2018 is $148.7 million , of which $54.0 million is included in the current portion of deferred revenue. As of December 31, 2018, the Company expected to recognize this amount over the remaining research term of the agreement, which is expected to extend through the first quarter of 2022 , with the majority being recognized through the fourth quarter of 2021. Alexion collaboration and equity agreements On October 22, 2018 , the Company and Alexion Pharma Holding Unlimited Company (“Alexion Pharma Holding”), an affiliate of Alexion Pharmaceuticals, Inc. (“Alexion Pharmaceuticals” and, together with Alexion Pharma Holding, “Alexion”) entered into a Collaborative Research and License Agreement (the “Alexion Collaboration Agreement”). The Alexion Collaboration Agreement is for the joint discovery and development of RNAi therapies for complement-mediated diseases. Under the terms of the Alexion Collaboration Agreement, the Company and Alexion will collaborate on the discovery and development of subcutaneously delivered GalXC candidates, currently in preclinical development, for the treatment of complement-mediated diseases with potential global commercialization by Alexion. The Company will lead the joint discovery and research efforts through the preclinical stage, and Alexion will lead development efforts beginning with Phase 1 studies. The Company will be responsible for manufacturing of the GalXC candidates through the completion of Phase 1, and the related costs will be paid by Alexion. Alexion will be solely responsible for the manufacturing of any product candidate subsequent to the completion of Phase 1. The Alexion Collaboration Agreement provides Alexion with exclusive worldwide licenses as well as development and commercial rights for two of the Company’s preclinical, subcutaneously delivered GalXC RNAi candidates and an exclusive option for the discovery and development of GalXC RNAi candidates against two additional complement pathway targets. Under the terms of the Alexion Collaboration Agreement, Alexion agreed to pay the Company a non-refundable, non-reimbursable, and non-creditable upfront payment of $22.0 million . The Alexion Collaboration Agreement also provides for potential additional payments to the Company of up to $600.0 million from proceeds from target option exercises and development and sales milestones, as defined in the agreement, which is comprised of: (i) option exercise fees of up to $20.0 million , representing $10.0 million for each of the targets selected; (ii) development milestones of up to $105.0 million for each product; and (iii) aggregate sales milestones of up to $160.0 million . Under the agreement, Alexion also agreed to pay to the Company mid-single to low-double digit royalties on potential product sales on a country-by-country, product-by-product basis until the later of the expiration of patent rights in a country, the expiration of market or regulatory exclusivity in such country, or 10 years after the first product sale in such country, subject to certain royalty step-down provisions set forth in the agreement. Simultaneously with the entry into the Alexion Collaboration Agreement, the Company and Alexion Pharmaceuticals entered into a Share Purchase Agreement (the “Alexion Share Issuance Agreement”), pursuant to which Alexion Pharmaceuticals purchased 835,834 shares of the Company’s common stock at $17.95 per share at issuance, for an aggregate stated purchase price of $15.0 million . Management concluded that the Alexion Share Issuance Agreement is to be combined with the Alexion Collaboration Agreement (together, the “Alexion Agreements”) for accounting purposes. With respect to the $15.0 million of cash received upon issuance of the shares, the Company applied equity accounting guidance to measure the $9.1 million recorded in equity upon the issuance of the shares, and the remaining $5.9 million was included as a component of the transaction price attributable to the revenue arrangement. Alexion selected two targets upon entry into the Alexion Collaboration Agreement, which, as noted above, provides Alexion with the option to select up to two additional targets, in exchange for an option fee payment of $10.0 million for each selected target. The Company concluded that Alexion is a customer in this arrangement, and as such, the element of the arrangement unrelated to the issuance of the shares falls within the scope of the revenue recognition guidance. The Company identified the following promises under the arrangement: (i) the granting of licenses of intellectual property and know-how rights; (ii) the option to select additional targets; (iii) the option to perform validation testing on additional targets; (iv) associated research and development services for the initial and, as applicable, additional targets; and (v) the Company’s participation in the joint steering committee. The Company concluded that the research and development services were not capable of being distinct from the research and development license, and were not distinct within the context of the contract, and should therefore be combined into a single performance obligation for each program. The Company considered the level of Alexion’s therapeutic expertise specifically related to RNAi, as well as Alexion’s know-how of the Company’s GalXC conjugates, and concluded that Alexion cannot currently benefit from the granted license on its own or together with other resources that are readily available to Alexion, including relationships with oligonucleotide vendors who synthesize GalXC conjugates under contract with the Company. As a result, the combination of the license of intellectual property together with the provision of research and development services together represent the highest level of goods and services that can be deemed distinct. Additionally, the Company determined that the options to select additional targets and to perform validation testing on additional targets were not priced at a discount and, as such, do not provide Alexion with material rights. Based on management’s assessments, the Company identified a single performance obligation, namely, the combined license and research and development services, for each of the two initially nominated targets. At the outset of the Alexion Collaboration Agreement, the transaction price was determined to be $37.4 million , which is comprised of the $22.0 million upfront payment, the $5.9 million identified upon issuance of the shares, as described above, and $9.5 million in aggregate contingent milestone payments that were either received or probable of achievement and under the Company’s control. The Company used the most likely amount method to estimate variable consideration and estimated that the most likely amount for each potential development milestone payment beyond the three initial research program milestones under this agreement was zero , as the achievement of those milestones is uncertain and highly susceptible to factors outside of the Company’s control. Accordingly, such milestones were excluded from the transaction price. Management will re-evaluate the transaction price at the end of each reporting period and as uncertain events are resolved or other changes in circumstances occur and adjust the transaction price as necessary. Sales-based royalties, including milestone payments based on the level of sales, were also excluded from the transaction price, as the license is deemed to be the predominant item to which the royalties relate. The Company will recognize such revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). Revenue associated with the performance obligations is being recognized as services are provided using an input method based on a cost-to-cost measure of progress method. The transfer of control occurs over time and, in management’s judgment, this input method is the best measure of progress towards satisfying the performance obligation and reflects a faithful depiction of the transfer of goods and services. During the year ended December 31, 2018 , the Company recognized $0.1 million as revenue under the Alexion Agreements in the accompanying consolidated statement of operations. The aggregate amount of the transaction price allocated to the Company’s partially unsatisfied performance obligations and recorded as deferred revenue at December 31, 2018 is $31.3 million , of which $11.7 million is included in current portion of deferred revenue. As of December 31, 2018, t he Company expects to recognize this amount over the remaining research program term, which is estimated to extend through the fourth quarter of 2023 , with the majority being recognized through the fourth quarter of 2021. BI Agreement and related amendments On October 27, 2017 , the Company entered into a collaborative research and license agreement with BI (the “BI Agreement”), pursuant to which the Company and BI jointly research and develop product candidates for the treatment of chronic liver disease using the GalXC platform, Dicerna’s proprietary RNAi-based technology. The BI Agreement is for the development of product candidates against one target gene with an option for BI to add the development of product candidates that target a second gene (the “Second Target”). Pursuant to the BI Agreement, Dicerna granted BI a worldwide license in connection with the research and development of such product candidates and transferred certain intellectual property rights of the selected product candidates to BI for clinical development and commercialization. Dicerna also may provide assistance to BI in order to help BI further develop selected product candidates. Under the terms of the BI Agreement, BI agreed to pay Dicerna a non-refundable upfront payment of $10.0 million for the first target, less a refundable withholding tax in Germany of $1.6 million . BI also agreed to reimburse Dicerna certain third-party expenses of $0.3 million . The German withholding tax was withheld by BI and remitted to the German tax authorities in accordance with local tax law. The Company received reimbursement of this tax in July 2018. During the term of the research program, BI will reimburse Dicerna the cost of certain materials and third-party expenses that have been included in the preclinical studies. The Company is eligible to receive up to $191.0 million in potential development and commercial milestones related to the initial target. Dicerna is also eligible to receive royalty payments on potential global net sales, subject to certain adjustments, tiered from high single digits up to low double-digits. BI’s Second Target option provided for an option fee payment of $5.0 million and success-based development and commercialization milestones and royalty payments to Dicerna. Milestone payments that are contingent upon the Company’s performance under the BI Agreement include potential developmental milestones totaling $99.0 million . The Company has excluded these amounts from allocable consideration at the outset of the arrangement, as described below. All potential net sales milestones, totaling $95.0 million , will be accounted for in the same manner as royalties and recorded as revenue at the later of the achievement of the milestone or the satisfaction of the performance obligation. The Company concluded that BI is a customer in this arrangement, and as such, the arrangement falls within the scope of the revenue recognition guidance. The Company identified the following performance obligations under the contract: the license of intellectual property and conducting agreed-upon research program services. The Company concluded that the license and research and development services are not capable of being distinct and are not distinct within the context of the contract; therefore, the Company considers these to be one performance obligation. The Company concluded the option underlying the transfer of future licenses and potential associated research for any not-yet-known target gene is not a performance obligation of the contract at inception because the option fee reflects the standalone selling price of the option, and therefore, the option is not considered to be a material right. The Company considered the level of BI’s therapeutic expertise specifically related to RNAi, as well as BI’s know-how with regard to the Company’s GalXC conjugates, and concluded that BI cannot currently benefit from the granted license on its own or together with other resources that are readily available to BI, including relationships with oligonucleotide vendors who synthesize GalXC conjugates under contract with the Company. As a result, the combination of the license of intellectual property together with the provision of research and development support services together represent the highest level of goods and services that can be deemed distinct. Based on management’s evaluation, the $10.0 million non-refundable upfront fee and the $0.3 million agreed-upon reimbursable third-party expenses constituted the amount of the consideration to be included in the transaction price and was allocated to the performance obligation identified. None of the development milestones have been included in the transaction price during the period, since none of such milestone amounts are within the control of the Company and are not considered probable to occur until confirmed by BI, at BI’s sole discretion. Any consideration related to commercial sales-based milestones (including royalties) will be recognized when the related sales occur, since these amounts have been determined to relate predominantly to the license granted to BI and therefore are recognized at the later of when the performance obligation is satisfied or when the related sales occur. The Company re-evaluates the transaction price in each reporting period and as uncertain events are resolved or other changes in circumstances occur and adjusts the transaction price as necessary. The $10.3 million transaction price is recognized over the research term, currently estimated to extend through August 2019 , which represents the Company’s current best estimate of the period of the obligation to provide research support services to BI. Related revenue is recognized on a straight-line basis, which is in management’s judgment an appropriate measure of progress toward satisfying the performance obligation, largely in absence of evidence that obligations are fulfilled in a specific pattern. BI contract amendment In October 2018, BI exercised its Second Target option, which entitles the Company to a non-refundable payment of $5.0 million upon the agreement of a research work plan and budget for the Second Target. The terms of the Second Target option exercise and related rights and obligations associated with the Second Target were agreed between the Company and BI in an Additional Target Agreement (the “ATA”), which was entered into on December 31, 2018 . Under the terms of the ATA, BI will be responsible for future clinical development and commercialization of candidate products for the Second Target. Additionally, during the term of the research program, BI will reimburse the Company for certain expenses. The Company is eligible to receive up to $170.0 million in potential development and commercial milestones related to the Second Target. The Company is also eligible to receive tiered royalty payments on potential global net sales, subject to certain adjustments, in the mid-single digits. Except as otherwise set forth in the ATA, development of the Second Target is subject to the terms of the original BI Agreement. Management determined that the addition of the Second Target upon exercise of the Second Target Option resulted in a new contract for accounting purposes, and the $5.0 million exercise price was representative of the standalone selling price. The exercise of the Second Target option on December 31, 2018 through the ATA created a new contract for accounting purposes. Consistent with the reasons described related to the initial target, management concluded that the non-refundable Second Target option exercise fee (akin to an upfront payment) constituted the amount of the consideration to be included in the transaction price and has been allocated to the single performance obligation. The basis for the conclusions regarding the treatment of development and sales-based milestones associated with the Second Target are consistent with those associated with the initial combined performance obligation under the BI Agreement. The Company will re-evaluate the transaction price in each reporting period and as uncertain events are resolved or other changes in circumstances occur. The $5.0 million transaction price , of which no revenue was recognized in 2018 as the program had not yet commenced, will be recognized over the research term, which is currently estimated to extend through June 2022 . In addition to establishing the terms of the Second Target option exercise, the ATA also amends the BI Agreement to provide BI with the option to add, over a three -year period, the development of product candidates targeting a further additional target gene (the “Third Target Option”). Per the ATA, if BI elects, in its sole discretion, to exercise the Third Target Option, the parties would agree to a research work plan and budget for the additional gene and negotiate development and commercialization milestones and royalty payments to the Company, and BI would make an option fee payment to the Company of $5.0 million . This option exercise fee is consistent with the Second Target option exercise fee, which management concluded was representative of the standalone selling price. If BI chooses to exercise the Third Target option, the Company will be responsible for the discovery and initial profiling of the product candidates, including primary preclinical studies, synthesis, and delivery. BI will be responsible for evaluating and selecting the product candidates for further development. If BI selects one or more product candidates, it will be responsible for further preclinical development, clinical development, manufacturing, and commercialization of those products. If the Third Target Option is exercised, such exercise would result in a new contract for accounting purposes, as the licensing rights and research and development services underlying the Third Target Option are distinct from those associated with the initial and Second Target. During the year ended December 31, 2018 , the Company recognized $6.1 million of revenue related to the BI Agreement, as amended, in the accompanying consolidated statement of operations. The aggregate amount of the transaction price allocated to the Company’s partially unsatisfied performance obligations and recorded as deferred revenue at December 31, 2018 is $3.2 million , all of which is included in current portion of deferred revenue. The Company expected to recognize this amount over the remaining research program term, which is eight months as of December 31, 2018 . The following table presents changes in the Company’s aggregate deferred revenue balances for each reporting period: YEAR ENDED BALANCE AT BEGINNING OF PERIOD ADDITIONS DEDUCTIONS BALANCE AT END OF PERIOD Deferred revenue, current and noncurrent $ 9,270 $ 180,092 $ (6,176 ) $ 183,186 YEAR ENDED BALANCE AT BEGINNING OF PERIOD ADDITIONS DEDUCTIONS BALANCE AT END OF PERIOD Deferred revenue, current and noncurrent $ — $ 10,300 $ (1,030 ) $ 9,270 The Company had no deferred revenue during the year ended December 31, 2016 . |
STOCKHOLDERS' EQUITY
STOCKHOLDERS' EQUITY | 12 Months Ended |
Dec. 31, 2018 | |
Equity [Abstract] | |
STOCKHOLDERS' EQUITY | STOCKHOLDERS' EQUITY Preferred stock The Company has authorized up to 5,000,000 shares of preferred stock, $0.0001 par value per share, for issuance. The preferred stock will have such rights, preferences, privileges and restrictions, including voting rights, dividend rights, conversion rights, redemption privileges and liquidation preferences, as shall be determined by the Company’s board of directors upon its issuance. At December 31, 2018 and 2017 , there were no shares of preferred stock outstanding. As further disclosed in Note 10 , during the year ended December 31, 2017 , the Company issued and sold in a private placement 700,000 shares of its newly designated redeemable convertible preferred stock, par value $0.0001 per share. Redeemable convertible preferred shares and the shares of common stock issuable upon conversion of the redeemable convertible preferred stock were offered and sold by the Company pursuant to an exemption from the registration requirements of the Securities Act provided by Section 4(a)(2) thereunder. On December 18, 2017, the Company completed the conversion of the redeemable convertible preferred stock and issued an aggregate of 24,206,663 shares of the Company’s common stock. Issuances of Common Stock On December 18, 2017 , the Company completed an underwritten follow-on public offering of 5,714,286 shares of common stock (the “2017 Offering”), which was made pursuant to the Company’s effective registration statement on Form S-3 previously filed with the SEC. In connection with the 2017 Offering, the Company entered into an underwriting agreement (the “2017 Underwriting Agreement”) with Stifel, Nicolaus & Company, Incorporated and Evercore Group LLC as representatives of the underwriters listed in the 2017 Underwriting Agreement (collectively, the “2017 Underwriters”), pursuant to which the Company granted to the 2017 Underwriters a 30 -day option to purchase up to an additional 857,143 shares of the Company’s common stock (the “Overallotment”). The Company completed the sale of 6,571,428 shares, inclusive of the Overallotment, to the 2017 Underwriters on December 18, 2017 , and that sale resulted in the receipt by the Company of aggregate gross proceeds of $46.0 million , less underwriter commissions and additional offering expenses totaling approximately $3.2 million . On April 20, 2018 , the Company entered into a Share Issuance Agreement with Alnylam (“Alnylam Share Issuance Agreement”), pursuant to which the Company agreed to issue to Alnylam 983,208 shares in satisfaction of the Company’s obligation under the Settlement Agreement to deliver shares to Alnylam (see Note 15 ). The Alnylam Share Issuance Agreement contains customary representations and warranties of each party. The transaction contemplated by the Alnylam Share Issuance Agreement was closed on April 24, 2018 . On September 11, 2018 , the Company completed an underwritten follow-on public offering of 7,680,492 shares of common stock (the “2018 Offering”). In connection with the 2018 Offering, the Company entered into an underwriting agreement (the “2018 Underwriting Agreement”) with Citigroup Global Markets Inc. and Leerink Partners LLC as representatives of the underwriters listed in the 2018 Underwriting Agreement (collectively, the “2018 Underwriters”), pursuant to which the Company granted to the 2018 Underwriters a 30 -day option to purchase up to an additional 1,152,073 shares of the Company’s common stock. The Company completed the sale of 8,832,565 shares to the 2018 Underwriters on September 11, 2018 ; the sale resulted in the receipt of gross proceeds of $115.0 million . In connection with the Alexion Collaboration Agreement, the Company and Alexion entered into the Alexion Share Issuance Agreement on October 22, 2018 , pursuant to which the Company sold to Alexion 835,834 shares of the Company’s common stock at $17.95 per share for an aggregate stated purchase price of approximately $15.0 million , of which $9.1 million was allocated to the share issuance for accounting purposes. Pursuant to the terms of the Alexion Share Issuance Agreement, Alexion may not, without the prior approval of the Company, dispose of any of the Alexion shares for a six-month period of time commencing on the closing date of the Alexion Share issuance. In connection with the Lilly Collaboration Agreement, the Company and Lilly entered into the Lilly Share Issuance Agreement on October 25, 2018 , pursuant to which the Company sold to Lilly 5,414,185 shares of common stock at $18.47 per share for an aggregate stated purchase price of approximately $100.0 million , of which $51.3 million was allocated to the share issuance for accounting purposes. T he closing of the transactions contemplated by the Lilly Collaboration Agreement and the Lilly Share Issuance Agreement occurred on December 19, 2018 . |
REDEEMABLE CONVERTIBLE PREFERRE
REDEEMABLE CONVERTIBLE PREFERRED STOCK | 12 Months Ended |
Dec. 31, 2018 | |
Temporary Equity Disclosure [Abstract] | |
REDEEMABLE CONVERTIBLE PREFERRED STOCK | REDEEMABLE CONVERTIBLE PREFERRED STOCK On April 11, 2017 , pursuant to a redeemable convertible preferred stock purchase agreement (“SPA”) with seven institutional investors (the “Preferred Holders”), led by funds advised by Bain Capital Life Sciences L.P. (“Lead Investor”), the Company issued and sold in a private placement 700,000 shares of its newly designated redeemable convertible preferred stock, par value $0.0001 per share, at a purchase price of $100.00 per share, for total gross proceeds of $70.0 million (“Private Placement”), less issuance costs of approximately $0.8 million . The redeemable convertible preferred shares and the shares of common stock issuable upon conversion of the redeemable convertible preferred stock were offered and sold by the Company pursuant to an exemption from the registration requirements of the Securities Act provided by Section 4(a)(2) thereunder. In addition to the Lead Investor, other participants in the Private Placement included affiliates of Cormorant Asset Management, LLC, Domain Associates, LLC (“Domain Associates”), EcoR1 Capital, LLC, RA Capital Management, LLC (“RA Capital”) and Skyline Management LLC (“Skyline Ventures”), among others. Domain Associates, RA Capital and Skyline Ventures are entities that are affiliated or were formerly affiliated with certain members of the Company’s board of directors. On March 28, 2017, in accordance with the terms of the SPA, the Company increased the size of its board of directors from eight to nine directors and approved the appointment of Adam M. Koppel, M.D., Ph.D., a managing director of the Lead Investor, as a director of the Company, effective as of the closing of the Private Placement on April 11, 2017. Dr. Koppel was reelected to the Company’s board of directors by shareholder vote in June 2017. The redeemable convertible preferred stock had the rights and preferences set forth in a Certificate of Designation, which was filed with the Secretary of State of the State of Delaware. Inducement and conversion On December 13, 2017, in connection with the 2017 Offering, defined and discussed in Note 9 , the Company entered into a letter agreement (the “Letter Agreement”) with the Preferred Holders. Pursuant to the Letter Agreement, the Preferred Holders agreed, subject to the completion of the 2017 Offering, to optionally convert all of their shares of redeemable convertible preferred stock, to the extent not subject to Conversion Blockers, into common stock, and consented, where applicable, to the repurchase of the residual shares of common stock that would have been issuable but for the Conversion Blockers (the “Residual Shares”) for $0.0001 per share. “Conversion Blockers” refers to the beneficial ownership limitations in the Company’s Certificate of Designation of the redeemable convertible preferred stock, which included (i) a 19.99% blocker provision to comply with Nasdaq Listing Rules, (ii) if so elected by a holder, a 9.99% blocker provision that would have prohibited beneficial ownership of more than 9.99% of the outstanding shares of the Company’s common stock or voting power at any time, and (iii) ownership limitations resulting from applicable regulatory restrictions. The Letter Agreement also provided for Preferred Holders to waive and amend certain provisions in an amended and restated registration rights agreement by and among the Company and the Preferred Holders party thereto (the “Registration Rights Agreement”). In consideration for the Preferred Holders’ agreeing to the optional conversion of the redeemable convertible preferred stock and to a waiver under and certain amendments to the Registration Rights Agreement, the Company agreed to issue to the Preferred Holders pre-funded warrants (the “Pre-Funded Warrants”), exercisable in part or in whole at any time upon grant for shares of the Company’s common stock at a price per share of $0.0001 per share. Each Preferred Holder was entitled to elect to receive shares of the Company’s common stock in lieu of the Pre-Funded Warrants that otherwise would have been issued to such Preferred Holder subject to any applicable Conversion Blockers. Under the Letter Agreement, the number of shares allocable to each Preferred Holder was calculated based on the sum of (i) the number of shares of common stock into which the additional dividend accruals on the redeemable convertible preferred stock that such Preferred Holders would have been entitled to receive up to and including March 31, 2018 would have been convertible, calculated immediately prior to the effectiveness of the conversion and (ii) any Residual Shares repurchased, or to be repurchased, from such Preferred Holder by the Company as described above (collectively, the “Additional Investor Shares”). The formula for the Additional Investor Shares assumes (1) a conversion price of $3.19 per share of common stock; (2) application of a dividend rate of 12% per annum from April 11, 2017 to October 27, 2017 and (3) application of a dividend rate of 8% per annum commencing from October 28, 2017 through March 31, 2018. On December 18, 2017, the Company completed the conversion of the redeemable convertible preferred stock and issued an aggregate of 24,206,663 shares of the Company’s common stock. No Pre-Funded Warrants were issued in connection with the conversion of the redeemable convertible preferred stock, as all Preferred Holders opted to receive common shares in lieu of Pre-Funded Warrants, largely given the inapplicability of Conversion Blockers as of the date of conversion, immediately after which no shares of redeemable convertible preferred stock remained outstanding. On December 29, 2017, the Company filed with the Secretary of State of the State of Delaware a Certificate of Elimination of the Redeemable Convertible Preferred Stock, which eliminates from the Company’s Certificate of Incorporation all matters set forth in the Certificate of Designation of Redeemable Convertible Preferred Stock previously filed with the Secretary of State of the State of Delaware, which established and designated the redeemable convertible preferred stock and the rights, powers, preferences, privileges and limitations thereof. Upon conversion of the redeemable convertible preferred stock, the Company applied the guidance outlined in the FASB’s Accounting Standard Codification (“ASC”) Topic 470-20, Debt with Conversion and Other Options (“ASC 470-20”), which contains guidance addressing the accounting for induced conversions of convertible debt, which in turn, per the U.S. Securities and Exchange Commission’s (“SEC”) guidance codified in ASC Topic 260, Earnings per Share (“ASC 260”), should be applied also to induced conversions of convertible preferred stock. The Company applied the guidance provided in ASC 260-10-S99-2 and compared the fair value of common stock transferred in the conversion transaction to the Preferred Holders to the fair value of common stock issuable pursuant to the original conversion terms. The resulting excess, which amounted to approximately $3.8 million , was recorded as a deemed dividend on conversion of the redeemable convertible preferred shares and has been added to net loss to arrive at net loss attributable to common stockholders in the accompanying consolidated statement of operations for the year ended December 31, 2017. Dividends Each holder of redeemable convertible preferred stock had been entitled to receive cumulative dividends on the Accrued Value, as defined below, of each share of redeemable convertible preferred stock at an initial rate of 12% per annum, compounded quarterly and subject to two rate reductions of 4% each in connection with the occurrence of one of the agreed-upon milestone events. Entering into the BI Agreement, as defined and discussed in Note 8 , constituted, per the Certificate of Designation, a milestone event for purposes of applying the first of two allowable rate reductions to dividends payable on the redeemable convertible preferred stock. As such, the dividend rate on the redeemable convertible preferred stock was reduced from 12% to 8% , effective on October 27, 2017. Dividends on the redeemable convertible preferred stock accrued on the Accrued Value of each share of redeemable convertible preferred until the conversion thereof, which occurred on December 18, 2017, as discussed above. “Accrued Value” meant, with respect to each share of redeemable convertible preferred stock, the sum of (i) $100.00 plus (ii) on each quarterly dividend date, an additional amount equal to the dollar value of any dividends on a share of redeemable convertible preferred stock which had accrued on any dividend payment date and had not previously been added to such Accrued Value. For accounting purposes, in accordance with ASC Topic 480-10-S99, Distinguishing Liabilities from Equity – SEC Materials (“ASC 480-10-S99”), the Company recorded the dividends at fair value at each dividend declaration date. The fair value of the dividends was determined using a binary lattice model that captured the intrinsic value of the underlying common stock on the declaration date and the option value of the shares and future dividends. The lattice model was used to determine fair value of dividends on each dividend date through September 30, 2017, which was the last dividend date prior to conversion of the redeemable convertible preferred shares, included the following inputs: JUNE 30, SEPTEMBER 30, Price per common share $ 3.17 $ 5.75 Expected term (in years) 6.75 6.50 Expected volatility 70.0 % 73.0 % Risk-adjusted discount rate 18.0 % 19.1 % In addition to the inputs presented above, use of the lattice model applied other assumptions, including probability simulations of various outcomes largely associated with the conversion-related milestone events referred to above and with the progression of the Company’s per common share price. Use of the lattice model resulted in a fair value estimate of the aggregate dividends declared on June 30, 2017 and September 30, 2017 of $1.9 million and $4.1 million , respectively. Beneficial conversion feature In accordance with ASC Topic 470-20, the Company recorded a beneficial conversion feature (“BCF”) related to the issuance of the redeemable convertible preferred. The BCF was recognized separately at issuance by allocating a portion of the proceeds equal to the intrinsic value of that feature to additional paid-in capital. The BCF was calculated at the commitment date, which management has determined to be the date of issuance. Intrinsic value is calculated as the difference between the effective conversion price and the fair value of the Company’s common stock, multiplied by the number of shares into which the issued shares of redeemable convertible preferred shares are convertible. During the year ended December 31, 2017, the Company recorded a deemed dividend charge of $6.1 million , to reflect full and immediate accretion of the discount resulting from the at-issuance BCF embedded within the redeemable convertible preferred stock as a result of the shares being immediately convertible into shares of the Company’s common stock at the option of the Preferred Holders. Accretion of the discount resulting from t he BCF and cumulative dividends, including accretion of share issuance costs, were non-cash transactions and have been reflected below net loss to arrive at net loss attributable to common stockholders. The following table reflects the changes in rede emable convertible preferred shares recorded during the year ended December 31, 2017: Balance at January 1, 2017 $ — Issuance of redeemable convertible preferred shares 70,000 Share issuance costs (750 ) Net proceeds 69,250 Discount resulting from the BCF at issuance (6,144 ) Accretion of the discount resulting from the BCF (deemed dividend) 6,144 Dividends accrued at the stated rates 5,515 Fair value in excess of dividends accrued at the stated rates 3,846 Accretion of share issuance costs (additional dividends) 750 Balance immediately prior to conversion 79,361 Conversion of redeemable convertible preferred shares (79,361 ) Balance at December 31, 2017 $ — |
STOCK-BASED COMPENSATION
STOCK-BASED COMPENSATION | 12 Months Ended |
Dec. 31, 2018 | |
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | |
STOCK-BASED COMPENSATION | STOCK-BASED COMPENSATION Equity Incentive Plans As of December 31, 2018 , the Company’s approved equity incentive plans include: the Third Amended and Restated 2007 Employee, Director and Consultant Stock Plan (“2007 Plan”); the 2010 Employee, Director and Consultant Equity Incentive Plan (“2010 Plan”); 2014 Employee Stock Purchase Plan (“2014 ESPP”); Amended and Restated 2014 Performance Incentive Plan (“2014 Plan”); and, the 2016 Inducement Plan (“2016 Plan”). These plans are administered by the board of directors and permit the granting of stock options, stock appreciation rights, stock bonuses, restricted stock, performance stock, stock units, phantom stock or similar rights to purchase or acquire shares. Upon adoption of the 2014 Plan, the Company no longer grants new equity awards under its 2007 Plan or 2010 Plan. Amended and Restated 2014 Performance Incentive Plan On January 14, 2014, the board of directors adopted 2014 Plan which authorized the issuance of up to 1,900,000 shares of the Company’s common stock, with an additional increase on the first trading day in January of each calendar year during the term of the plan by an amount equal to 4% of the total number of shares of Common Stock issued and outstanding on December 31 of the immediately preceding calendar year. In June 2015, the 2014 Plan was amended to increase the replenishment percentage from 4% to 5% of outstanding common shares annually and to allow the reissuance thereunder of awards and grants that expire or are canceled, terminated, forfeited or fail to vest under the 2007 Plan and 2010 Plan, as amended. Stock options for new hires granted under this plan generally vest 25% after 12 months, followed by ratable vesting over the remaining 36 -month term and expire 10 years from the grant date. Annual promotional and incentive-related grants generally vest ratably over a period of 48 months. As of December 31, 2018 , there were 872,411 shares of common stock reserved for future issuance under the 2014 Plan. Inducement Grants During 2014 and 2015, the Company granted 470,272 and 450,700 stock options, respectively, as an inducement material to individuals entering into employment with the Company (“Inducement Grants”). The Inducement Grants were approved by the Compensation Committee of the Company’s board of directors and were awarded in accordance with Nasdaq Listing Rule 5635(c)(4) and outside of the 2014 Plan. As such, any shares underlying the Inducement Grants are not, upon forfeiture, cancellation or expiration, returned to a pool of shares reserved for future issuance. As of December 31, 2018 , there were 130,000 Inducement Grants that remained outstanding. 2016 Inducement Plan On March 4, 2016, the board of directors adopted the 2016 Plan pursuant to which the Company may grant options to purchase common shares as an inducement to individuals to join the Company. The 2016 Plan, as adopted, allowed the Company to deliver up to 250,000 shares (the “Share Limit”) of its common stock to eligible persons, as defined. The Share Limit is subject to adjustment as contemplated by the provisions of the 2014 Plan. In February and May 2017, the Share Limit was adjusted to increase the pool of issuable options by 125,000 and 200,000 underlying shares, respectively. On December 11, 2018, the board of directors approved a resolution to further increase the Share Limit under the 2016 Plan by 2,700,000 to 3,275,000 underlying shares. There were no stock options granted pursuant to the 2016 Plan during the year ended December 31, 2018 . As of December 31, 2018 , the Company has 2,875,000 shares of common stock reserved for future issuance under the 2016 Plan. Stock-based compensation expense The Company has classified stock-based compensation expense in its consolidated statements of operations as follows: YEAR ENDED DECEMBER 31, 2018 2017 2016 Research and development expenses $ 3,062 $ 3,536 $ 4,467 General and administrative expenses 4,826 4,234 4,698 Total $ 7,888 $ 7,770 $ 9,165 Stock options Expected volatility for the Company’s common stock was determined based on an average of the historical volatility of a peer group of similar companies due to limited historical volatility of the Company’s own common stock. The Company also has limited stock option exercise information, and as such, the expected term of stock options granted was calculated in most cases using the simplified method, which represents the average of the contractual term of the stock option and the weighted average vesting period of the stock option. The assumed dividend yield is based upon the Company’s expectation of not paying dividends in the foreseeable future. The risk-free rate for periods within the expected life of the stock option is based upon the U.S. Treasury yield curve in effect at the time of grant. The assumptions used in the Black-Scholes option-pricing model for all stock options granted during each period presented are as follows: YEAR ENDED DECEMBER 31, 2018 2017 2016 Common stock price $9.14 - $15.74 $2.49 – $9.71 $2.94 – $9.09 Expected option term (in years) 5.50 - 6.25 5.50 – 6.25 5.50 – 6.25 Expected volatility 75.9% - 78.3% 79.4% – 91.1% 70.9% – 79.4% Risk-free interest rate 2.3% - 3.0% 1.9% – 2.2% 1.2% – 2.0% Expected dividend yield 0.0% 0.0% 0.0% The table below summarizes the activity under the Company’s equity incentive plans: NUMBER WEIGHTED- EXERCISE WEIGHTED- AGGREGATE INTRINSIC VALUE OUTSTANDING – JANUARY 1, 2018 6,124,096 $ 8.58 Granted 2,241,350 $ 11.07 Exercised (329,934 ) $ 5.31 Forfeited/Canceled (90,026 ) $ 5.71 Expired (157,796 ) $ 14.08 OUTSTANDING – DECEMBER 31, 2018 7,787,690 $ 9.36 7.3 $ 24,305 EXERCISABLE – DECEMBER 31, 2018 4,842,084 $ 9.71 6.4 $ 16,151 VESTED AND EXPECTED TO VEST – DECEMBER 31, 2018 7,567,801 $ 9.30 7.2 $ 23,948 The weighted average grant date fair value of stock options granted during the years ended December 31, 2018 , 2017 , and 2016 was $7.64 , $2.52 , and $4.60 per share, respectively. As of December 31, 2018 , there was $17.1 million of unrecognized compensation cost related to unvested employee stock options which are expected to be recognized over a weighted average period of 2.9 years. The intrinsic value of stock options exercised was $2.9 million , $0.1 million , and $0.2 million for the years ended December 31, 2018 , 2017 , and 2016 , respectively. The Company does not currently hold any treasury shares. Upon stock option exercise, the Company issues new shares and delivers them to the participant. Restricted common stock In 2014, the Company issued a total of 44,000 shares of the Company’s restricted common stock, of which 4,000 shares were fully vested at the grant date and the remaining shares were scheduled to vest in equal tranches over a four -year period on the anniversary date of the related grant. The fair value of these shares totaled $0.7 million at the grant date, representing a weighted average grant date fair value per share of $16.30 . At December 31, 2017, there were 10,000 shares of the Company’s restricted common stock remaining outstanding with a weighted average grant date fair value of $16.30 . During the year ended December 31, 2018, all 10,000 shares of restricted common stock with a weighted average grant date fair value of $16.30 vested and there are no outstanding shares of restricted common stock at December 31, 2018 . The total fair value of restricted common stock vested during the years ended December 31, 2018 and 2016 was $0.1 million and $0.1 million , respectively. The total fair value of restricted common stock that vested during the year ended December 31, 2017 was immaterial. Common stock warrants At December 31, 2017, the Company had 87,901 common stock warrants outstanding with a weighted average exercise price of $13.80 . All of the Company’s outstanding common stock warrants have been exercisable since November 30, 2013. During the year ended December 31, 2018, certain warrant holders exercised warrants to purchase 85,703 shares of the Company’s common stock on a net basis and received 45,710 shares of common stock and 39,993 shares were used to cover the exercise price of $7.00 per share. At December 31, 2018, there were 2,198 common stock warrants remaining outstanding with an exercise price of $250.00 per share and a remaining contractual life of 1.46 years . Employee stock purchase plan On January 28, 2014, the Company’s stockholders approved the 2014 ESPP, which authorized the issuance of up to 1,000,000 shares of common stock thereunder. The 2014 ESPP provides for an automatic reserve increase equivalent to the lesser of 1% of the total number of shares of common stock issued and outstanding on December 31 of the immediately preceding calendar year and 1,000,000 shares of common stock, unless otherwise determined by the Company’s board of directors. As of December 31, 2018 , there were 2,107,791 shares of common stock authorized and 1,845,179 shares of common stock available for issuance under the 2014 ESPP. Eligible employees may purchase shares of the Company’s common stock through regular payroll deductions up to 15% of their eligible compensation. Under the terms of the offering under the 2014 ESPP, the number of shares purchased by an individual participant in the plan may not exceed 10,000 shares in any one purchase period. In addition, the fair market value of shares purchased by an individual participant in the plan may not exceed $25,000 if the contribution period is within any one calendar year. Participants are allowed to terminate their participation in the ESPP at any time during the purchase period prior to the purchase of the shares. The offering periods have a 24 ‑month term; which consists of four purchase periods, each of which is six months in duration. New offering periods commence on the first day of January and July each year and end on the last business day of the immediately following June or December, respectively. The per-share purchase price at the end of each offering period is equal to the lesser of 85% of the fair market value of the common stock on the grant date of the offering period to which the purchase period relates or 85% of the fair market value of the common stock on the purchase date of the applicable purchase period. In the event that the fair value of the common stock on any purchase date during an offering period is lower than the fair market value of the common stock on the grant date of that offering period, that offering period will terminate on such purchase date, and each participant in such terminated offering period will be automatically enrolled in the new offering period that commences on the first business day of the next offering period that immediately follows such purchase date. Shares issued under the 2014 ESPP are considered compensatory. Accordingly, the Company is required to measure the fair value of the stock purchase rights granted and record compensation expense for share purchase rights granted under the 2014 ESPP. The fair values of the stock purchase rights are estimated using the Black-Scholes option-pricing model, which relies on a number of key assumptions to in calculating the estimates of fair value. Stock-based compensation expense related to stock purchase rights under the 2014 ESPP was $0.1 million , $0.4 million , $0.4 million and for the years ended December 31, 2018 , 2017 , and 2016 . During the years ended December 31, 2018 , 2017 , and 2016 , the Company issued 118,239 , 84,890 , and 36,501 shares of common stock under the 2014 ESPP, respectively. The weighted average purchase price of shares issued under the 2014 ESPP were $2.61 , $2.45 , and $4.53 per share for the years ended December 31, 2018 , 2017 , and 2016 , respectively. |
401(K) PROFIT SHARING PLAN AND
401(K) PROFIT SHARING PLAN AND TRUST | 12 Months Ended |
Dec. 31, 2018 | |
Retirement Benefits [Abstract] | |
401(K) PROFIT SHARING PLAN AND TRUST | 401(K) PROFIT SHARING PLAN AND TRUST The Company has a 401(k) Profit Sharing Plan and Trust (“401(k) Plan”), which is a retirement plan in which substantially all employees are eligible to participate. Eligible employees may elect to contribute up to the maximum limits, as set by the Internal Revenue Service, of their eligible compensation. Under the terms of the 401(k) Plan, employees may elect to make pre-tax and Roth contributions through payroll deductions within statutory and plan limits. The Company makes matching contributions of 300% of eligible employee salary deferrals that do not exceed 2% of the eligible participant’s compensation. All matching contributions vest immediately. Each year, the Company may also make a discretionary profit sharing contribution to the plan. Such contributions to the Plan are allocated among eligible participants in the proportion of their salaries to the total salaries of all participants. Expense recognized by the Company for matching contributions made to the 401(k) Plan was $0.6 million , $0.4 million , and $0.2 million for the years ended December 31, 2018 , 2017 , and 2016 , respectively. There were no discretionary profit sharing contributions made by the Company during the years ended December 31, 2018 , 2017 , or 2016 . |
INCOME TAXES
INCOME TAXES | 12 Months Ended |
Dec. 31, 2018 | |
Income Tax Disclosure [Abstract] | |
INCOME TAXES | INCOME TAXES The Company has no current and no deferred income tax expense for the years ended December 31, 2018 and 2017 , respectively. The Company did no t record a federal income tax provision or benefit for the years ended December 31, 2018 , 2017 , and 2016 , respectively. The reconciliation between income taxes computed at the federal statutory income tax rate and the provision for (benefit from) income taxes is as follows: YEAR ENDED DECEMBER 31, 2018 2017 2016 Federal statutory rate 21.0 % 34.0 % 34.0 % Effect of: Foreign rate differential (9.5 )% (17.6 )% (31.4 )% Tax reform — % (29.6 )% — % Net operating loss limitation (23.0 )% — % — % Change in valuation allowance 10.6 % 13.5 % — % Research and development tax credit 0.5 % 0.6 % (0.7 )% Stock-based compensation expense 0.4 % (0.8 )% (0.9 )% Other — % (0.1 )% (1.0 )% Total — % — % — % The components of the Company’s deferred tax assets are as follows: DECEMBER 31, 2018 2017 Deferred tax assets: Net operating loss carryforwards $ 24,739 $ 32,008 Capitalized research and development costs 516 618 Research and development credit carryforwards 3,988 3,481 Stock-based compensation expense 7,644 6,066 Depreciation expense and other costs 75 42 Net deferred tax assets 36,962 42,215 Valuation allowance (36,962 ) (42,215 ) Net deferred tax assets $ — $ — On December 22, 2017, the Tax Cuts and Jobs Act (the “TCJA”) was signed into law in the United States. The TCJA reduced the U.S. corporate tax rate from 34% to 21% for tax years beginning after December 31, 2017. As a result of the newly enacted law, the Company was required to revalue all deferred tax assets and liabilities existing as of December 31, 2017 so as to reflect the reduction in the federal tax rate. This revaluation resulted in a reduction to the Company’s deferred tax asset of $17.8 million at December 31, 2017, with a corresponding reduction to the Company’s valuation allowance. Consequently, there was no impact on the accompanying consolidated financial statements that resulted from the reduction in the federal tax rate. Other relevant provisions of the TCJA did not have a material impact on the accompanying consolidated financial statements. Management has evaluated the positive and negative evidence bearing upon the realizability of the Company’s net deferred tax assets and has determined that it is more likely than not that the Company will not recognize the benefits of the net deferred tax assets. As a result, the Company has recorded a full valuation allowance at December 31, 2018 and 2017 . Realization of the future tax benefits is dependent on many factors, including the Company’s ability to generate taxable income within the net operating loss carryforward period. Under the provisions of the Internal Revenue Code, certain substantial changes in the Company’s ownership, including a sale of the Company or significant changes in ownership due to sales of equity, may have limited, or may limit in the future, the amount of net operating loss carryforwards which could be used annually to offset future taxable income. At this time, there is an estimated limitation of approximately $97.0 million of net operating losses. As of December 31, 2018 , the Company had approximately $158.7 million of federal and $148.2 million of state net operating loss carryforwards. If not utilized, the federal and state net operating loss carryforwards expire starting in 2028 and 2030 , respectively. Additionally, as of December 31, 2018 , the Company had $2.6 million of federal and $1.4 million of Massachusetts tax credits that expire starting in 2028 and 2023 , respectively. As of December 31, 2018 , the Company had $1.6 million of unrecognized tax benefits, all of which would affect income tax expense if recognized, before consideration of the Company’s valuation allowance. The Company does not expect the unrecognized tax benefits to change significantly over the next 12 months . The Company recognizes both interest and penalties associated with uncertain tax positions as a component of income tax expense. As of December 31, 2018 and 2017 , the Company had no accrued penalties or provisions for interest. A reconciliation of the gross unrecognized tax benefit is as follows: YEAR ENDED DECEMBER 31, 2018 2017 Unrecognized tax benefits at the beginning of the period $ 1,451 $ 1,210 Additions for current tax positions 211 243 Changes for previous tax positions (31 ) (2 ) Unrecognized tax benefits at the end of the period $ 1,631 $ 1,451 The Company files income tax returns in the United States, the Commonwealth of Massachusetts, Colorado, and New Jersey. The tax years 2008 through 2017 remain open to examination by these jurisdictions, as carryforward attributes generated in past years may be adjusted in a future period. The Company is not currently under examination by the Internal Revenue Service or any other jurisdiction for these years. The Company has not recorded any interest or penalties for unrecognized tax benefits since its inception. |
COMMITMENTS AND CONTINGENCIES
COMMITMENTS AND CONTINGENCIES | 12 Months Ended |
Dec. 31, 2018 | |
Commitments and Contingencies Disclosure [Abstract] | |
COMMITMENTS AND CONTINGENCIES | COMMITMENTS AND CONTINGENCIES Facility lease On July 11, 2014, the Company executed a non-cancelable operating lease for office and laboratory space in Cambridge, Massachusetts. The lease agreement, the term of which commenced on December 1, 2014 , obligates the Company to make minimum payments totaling $9.6 million over a six -year lease term ending November 30, 2020 . The Company has the option to extend the lease term for one additional five -year period. Rent expense is recorded on a straight-line basis. As part of the lease agreement, the Company established a letter of credit, secured by a restricted money market account, the balance of which is presented as restricted cash equivalents at December 31, 2018 and 2017 . Future minimum lease payments on the Company’s non-cancelable operating lease for office and laboratory space are as follows: YEARS ENDING DECEMBER 31, OPERATING LEASE 2019 $ 1,678 2020 1,581 Total $ 3,259 Rent expense was $1.6 million for each of the years ended December 31, 2018 , 2017 , and 2016 . Legal proceedings From time to time, the Company may be subject to various claims and legal proceedings. If the potential loss from any claim, asserted or unasserted, or legal proceeding is considered probable and the amount is reasonably estimable, the Company will accrue a liability for the estimated loss. There were no contingent liabilities recorded as of December 31, 2018 or 2017 . |
LITIGATION
LITIGATION | 12 Months Ended |
Dec. 31, 2018 | |
Commitments and Contingencies Disclosure [Abstract] | |
LITIGATION | LITIGATION On June 10, 2015 , Alnylam filed a complaint against the Company in the Superior Court of Middlesex County, Massachusetts. The complaint alleged misappropriation of confidential, proprietary, and trade secret information, as well as other related claims, in connection with the Company’s hiring of a number of former employees of Merck & Co., Inc. (“Merck”) and its discussions with Merck regarding the acquisition of its subsidiary, Sirna Therapeutics, Inc., which was subsequently acquired by Alnylam. On April 18, 2018 , the Company and Alnylam entered into the Settlement Agreement, resolving all ongoing litigation between the Company and Alnylam. The terms of the Settlement Agreement include mutual releases and dismissals with prejudice of all claims and counterclaims in the following litigation between the parties: (i) Alnylam Pharmaceuticals, Inc. v. Dicerna Pharmaceuticals, Inc., No. 15-4126 pending in the Massachusetts Superior Court for Middlesex County and (ii) Dicerna Pharmaceuticals, Inc., v. Alnylam Pharmaceuticals, Inc. No.1:17-cv-11466 pending in the United States District Court for the District of Massachusetts. Pursuant to the terms of the Settlement Agreement, the Company has agreed to make the following payments to Alnylam: (i) a $2.0 million upfront payment in cash, which the Company made in May 2018 ; (ii) an additional $13.0 million in cash to be paid as 10.0% of any upfront or first year cash consideration that the Company receives pursuant to future collaborations related to Ga1NAc-conjugated RNAi research and development (excluding any amounts received or to be received by the Company from its existing collaboration with BI), provided that the $13.0 million must be paid by no later than April 28, 2022 ; and (iii) issuance of shares of the Company’s common stock pursuant to the Alnylam Share Issuance Agreement. Under the Settlement Agreement, for periods ranging from 18 months up to four years , the Company will be restricted in its development and other activities relating to oligonucleotide-based therapeutics directed toward a defined set of eight Alnylam targets (the “Oligo Restrictions”). The Oligo Restrictions pertain to targets where Dicerna does not have, or does not currently intend to have, a therapeutic program, or are expected to be consistent with Dicerna’s execution on programs in the normal course of business. The Settlement Agreement did not include any admission of liability or wrongdoing by either party or any licenses to any intellectual property from either party. On April 20, 2018 , the Company and Alnylam entered into the Alnylam Share Issuance Agreement, pursuant to which the Company agreed to issue to Alnylam 983,208 shares in satisfaction of the Company’s obligation under the Settlement Agreement to deliver shares to Alnylam. The 983,208 shares issued pursuant to the Alnylam Share Issuance Agreement was recorded at fair market value of $10.3 million based on the Company’s closing share price on April 18, 2018 , the date the Settlement Agreement was executed. The Company did not assign any value to the Oligo Restrictions as the Company did not incur additional losses or give up any value as a result of the restrictions. In May 2018 , the Company recorded the cash obligation of $13.0 million as a liability discounted to the estimated present value of $8.7 million at an effective interest rate of 10.0% . The Company applied the effective interest method, as the present value is accreted through maturity. In October 2018, the Company entered into collaboration agreements with Alexion and Lilly, under which the Company is entitled to upfront cash consideration of $22.0 million and $100.0 million , respectively (see Note 8 ). Accordingly, the Company revised its estimate of the present value of the litigation settlement payable from $8.7 million to $13.0 million based on the expected timing of the remaining payments. The impact of revising the expected timing of repayment was recorded as a $3.7 million charge to litigation expense in the consolidated statement of operations for the year ended December 31, 2018 . In connection with the execution of the Alexion Collaboration Agreement and related the receipt the non-refundable, non-reimbursable, and non-creditable upfront payment of $22.0 million and proceeds of $15.0 million from the Alexion Share Issuance Agreement in October 2018 , the Company determined that $2.5 million became payable to Alnylam under the terms of the Settlement Agreement. The Company issued a payment to Alnylam of $2.5 million in November 2018 for the amount of the litigation settlement payable due in connection with the cash consideration received from Alexion during 2018 . At December 31, 2018 , the outstanding balance of the litigation settlement payable was $10.5 million . The Company paid the remaining outstanding balance of litigation settlement payable in full on January 22, 2019 . During the year ended December 31, 2018 , the Company recognized interest expense of $0.6 million on the outstanding balance of the litigation settlement payable during the year. Total litigation expense was $29.1 million for the year ended December 31, 2018 , all of which related to the litigation and settlement agreement with Alnylam. The litigation expense for the year ended December 31, 2018 includes $24.7 million related to the Settlement Agreement. The Company recorded expenses related to the Alnylam litigation of $9.0 million during the year ended December 31, 2017 . |
QUARTERLY FINANCIAL DATA (UNAUD
QUARTERLY FINANCIAL DATA (UNAUDITED) | 12 Months Ended |
Dec. 31, 2018 | |
Quarterly Financial Information Disclosure [Abstract] | |
QUARTERLY FINANCIAL DATA (UNAUDITED) | QUARTERLY FINANCIAL DATA (UNAUDITED) The following tables contain selected quarterly financial information for the years ended December 31, 2018 and 2017 . The Company believes that the following information reflects all normal recurring adjustments necessary for a fair statement of the information for the periods presented. 2018 FIRST QUARTER SECOND QUARTER THIRD QUARTER FOURTH QUARTER TOTAL YEAR Revenue from collaborative arrangements $ 1,545 $ 1,545 $ 1,545 $ 1,541 $ 6,176 Net loss $ (15,579 ) $ (35,644 ) $ (19,020 ) $ (18,610 ) $ (88,853 ) Net loss attributable to common stockholders $ (15,579 ) $ (35,644 ) $ (19,020 ) $ (18,610 ) $ (88,853 ) Net loss per share attributable to common stockholders – basic and diluted $ (0.30 ) $ (0.68 ) $ (0.35 ) $ (0.29 ) $ (1.60 ) 2017 FIRST QUARTER SECOND QUARTER THIRD QUARTER FOURTH QUARTER TOTAL YEAR Revenue from collaborative arrangements $ — $ — $ — $ 1,030 $ 1,030 Net loss $ (14,201 ) $ (15,225 ) $ (15,033 ) $ (15,741 ) $ (60,200 ) Net loss attributable to common stockholders $ (14,201 ) $ (23,991 ) $ (19,144 ) $ (22,956 ) $ (80,292 ) Net loss per share attributable to common stockholders – basic and diluted $ (0.68 ) $ (1.15 ) $ (0.92 ) $ (0.91 ) $ (3.66 ) Net loss per share attributable to common stockholders is based on each reporting period’s weighted ‑ average number of shares outstanding, which may differ on a quarter-to-quarter basis. As such, the sum of the quarterly net loss per share attributable to common stockholders may not equal the year-to-date net loss per share attributable to common stockholders. |
SUBSEQUENT EVENTS
SUBSEQUENT EVENTS | 12 Months Ended |
Dec. 31, 2018 | |
Subsequent Events [Abstract] | |
SUBSEQUENT EVENTS | SUBSEQUENT EVENTS Lexington lease On January 2, 2019, the Company entered into a non-cancelable real property lease agreement with Hayden Office Trust under a Declaration of Trust dated August 24, 1977, as the same may have been amended, for approximately 80,872 square feet of laboratory and office space in Lexington, Massachusetts (the “Lexington Lease”). The Company intends to move its corporate headquarters and research facility to this location upon occupancy, which is expected to occur in the fourth quarter of 2019. The original term (the “Original Term”) of the Lexington Lease is seven years, commencing on the earlier of (a) the date on which the premises are ready for occupancy under the terms of the lease, or (b) the date on which the Company commences occupancy of any portion of the premises for the permitted uses under the lease. The Company has options to extend the term of the lease for two additional successive periods of five years each (the “Extension Periods”). Annual fixed rent will be approximately $3.9 million for the first 12-month period during the Original Term, increasing on an annual basis until reaching approximately $4.7 million for the seventh 12-month period during the Original Term. The Lexington Lease provides for an aggregate fixed rent of approximately $30.1 million during the seven -year Original Term. Annual fixed rent during the Extension Periods will be agreed upon between the Company and the Landlord following the Company’s provision of notice of its intention to exercise an extension option. If the Company and the Landlord cannot agree on annual fixed rent during an Extension Period, the Company will have the right to seek, subject to the terms of the Lexington Lease, a broker determination of the prevailing market rent, and the annual fixed rent during such Extension Period will be the prevailing market rent determined by the broker. In addition to the annual fixed rent, the Company will be responsible for certain customary operating expenses and real estate taxes specified in the agreement. The Lexington Lease also contains customary default provisions allowing the landlord to terminate the lease or seek damages if the Company fails to cure certain breaches of its obligations under the lease within specified periods of time. In addition, the Company will be obligated to indemnify the landlord for certain losses incurred in connection with the Company’s use or occupancy of the premises. Cambridge sublease On January 4, 2019, the Company entered into a non-cancelable real property sublease agreement with PPF OFF 150 Cambridge Park Drive, LLC (the “Landlord”) and International Business Machines Corporation (the “Sublandlord”), for approximately 9,653 square feet of office space in Cambridge, Massachusetts (“Cambridge Sublease”). The term of the sublease commenced on January 11, 2019, the date that the Landlord provided written consent to the Cambridge Sublease, and extends through the sublease expiration date of July 30, 2021. The Cambridge Sublease provides for an aggregate fixed rent of approximately $0.8 million during the term of the sublease. |
SUMMARY OF SIGNIFICANT ACCOUN_2
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Policies) | 12 Months Ended |
Dec. 31, 2018 | |
Accounting Policies [Abstract] | |
Business | Business Dicerna ™ Pharmaceuticals, Inc. (“Dicerna” or the “Company”), a Delaware corporation founded in 2006 and headquartered in Cambridge, Massachusetts, is a biopharmaceutical company focused on the discovery and development of innovative subcutaneously delivered ribonucleic acid (“RNA”) interference (“RNAi”)-based pharmaceuticals using its GalXC ™ RNAi platform for the treatment of diseases involving the liver, including rare diseases, viral infectious diseases, chronic liver diseases, and cardiovascular diseases. Within these therapeutic areas, the Company believes its GalXC RNAi platform will allow the Company to build a broad pipeline of therapeutics with attractive pharmaceutical properties, including a subcutaneous route of administration, infrequent dosing (e.g., dosing that is monthly or quarterly, and potentially even less frequent), high therapeutic index, and specificity to a single target gene. |
Basis of presentation | Basis of presentation The accompanying consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America (“GAAP”) and include the accounts of Dicerna Pharmaceuticals, Inc. and its wholly-owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. |
Reclassifications | Reclassifications Effective January 1, 2018, the Company changed the presentation of the income from government grants from the caption “Grant revenue” to an offset to research and development expenses. Also, effective April 1, 2018, the Company changed the presentation of certain litigation-related expenses associated with the litigation with Alnylam Pharmaceuticals, Inc. (“Alnylam”) from the caption “General and administrative” expense to “Litigation expense.” The changes associated with changes in presentation were applied retrospectively through the recast of affected prior period amounts in the consolidated statements of operations. The primary effects of such changes were: • the reclassification of grant income from revenue to the presentation as an offset to research and development expenses of $1.1 million and $0.3 million for the years ended December 31, 2017 and 2016, respectively; and • the reclassification of certain litigation-related expenses historically included in general and administrative expense to litigation expense in the consolidated statements of operations of $9.0 million and $2.9 million for the years ended December 31, 2017 and 2016, respectively. |
Significant judgments and estimates | Significant judgments and estimates The preparation of consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, and the disclosure of contingent assets and liabilities at the date of the consolidated financial statements, as well as the revenues and expenses incurred during the reporting periods. On an ongoing basis, the Company evaluates its judgments and estimates, including those related to revenue recognition and accrued expenses. The Company bases its estimates on historical experience and on various other factors that the Company believes are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not apparent from other sources. Changes in estimates are reflected in reported results for the period in which they become known. Actual results could differ materially from those estimates. |
Cash and cash equivalents | Cash and cash equivalents Cash and cash equivalents includes all highly liquid investments, including money market funds, maturing within 90 days from the date of purchase. |
Restricted cash equivalents | Restricted cash equivalents Restricted cash equivalents includes the balance of funds held in a money market collateral account that is restricted to secure a letter of credit for the Company’s operating lease for office and laboratory space at 87 Cambridgepark Drive in Cambridge, Massachusetts. The letter of credit is required to be maintained throughout the term of the Company’s lease, which expires on November 30, 2020 . |
Concentrations of credit risk and significant customers | Concentrations of credit risk and significant customers Financial instruments that subject the Company to significant concentrations of credit risk consist of cash, cash equivalents, restricted cash equivalents, held-to-maturity investments, contract receivables, and the withholding tax receivable (see Note 8 – Collaborative Research and License Agreements). All of the Company’s cash, cash equivalents, restricted cash equivalents, and held-to-maturity investments are invested in money market funds or United States (“U.S.”) treasury securities that management believes to be of high credit quality. The Company’s revenues for the year ended December 31, 2018 are a result of the Company’s collaboration agreements with Boehringer Ingelheim (“BI”) and Alexion Pharmaceuticals, Inc. (“Alexion”). BI represented substantially all of the Company’s revenue from collaborative arrangements for the years ended December 31, 2018 and 2017 . All revenues recognized by the Company to date were earned in the U.S. At December 31, 2018 , the balance of the Company’s contract receivables was solely related to the non-refundable, non-creditable upfront payment due to the Company in connection with a collaboration agreement entered into with Lilly (see Note 8 – Collaborative Research and License Agreements ). The Company did no t have any contract receivables at December 31, 2017 . The Company does not currently own or operate any manufacturing facilities for the production of preclinical, clinical, or commercial quantities of any of its product candidates. For each product candidate, the Company currently contracts with manufacturers, and expects to continue to do so to meet the preclinical and clinical requirements of its product candidates. |
Property and equipment | Property and equipment Property and equipment are stated at cost. Major betterments are capitalized whereas expenditures for maintenance and repairs which do not improve or extend the life of the respective assets are charged to operations as incurred. Depreciation is provided using the straight-line method over the estimated useful lives, as shown below: ASSET CATEGORY ESTIMATED Laboratory equipment 5 years Office and computer equipment 3 - 5 years Furniture and fixtures 5 years Leasehold improvements 5 years or the remaining Construction in process is stated at cost, which includes the cost of construction and other direct costs attributable to the construction. No provision for depreciation and amortization expense is recorded related to construction in process until the relevant assets are completed and put into use. At December 31, 2018 , the balance of construction in process includes costs associated with laboratory equipment under installation and the construction of certain leasehold improvements. |
Impairment of long-lived assets | Impairment of long-lived assets Long-lived assets are reviewed for impairment whenever events or changes in circumstances indicate that the carrying amount of the asset may not be recoverable. When such events occur, the Company compares the carrying amounts of the assets to their undiscounted expected future cash flows. If this comparison indicates that there is an impairment, the amount of the impairment is calculated as the difference between the carrying value and fair value of the related asset. During the years ended December 31, 2018 , 2017 , and 2016 , no impairments were recorded. |
Segment and geographic information | Segment and geographic information Operating segments are defined as components (business activity from which it earns revenue and incurs expenses) of an enterprise about which discrete financial information is available and regularly reviewed by the chief operating decision maker in deciding how to allocate resources and in assessing performance. The Company, through its Chief Executive Officer in his role as chief operating decision maker, views its operations and manages its business as one operating segment. All long-lived assets of the Company are located in the United States. |
Research and development costs | Research and development costs Research and development costs consist of expenses incurred in performing research and development activities, including compensation and benefits for full-time research and development employees, an allocation of facility expenses, overhead expenses and other outside expenses. Research and development costs are expensed as incurred. Research and development costs that are paid in advance of performance are deferred as a prepaid expense and amortized over the service period as the services are provided. The Company records grants from governmental and non-profit agencies as a reduction in research and development expense. Grants are recognized when there is reasonable assurance that the Company will comply with the conditions attached to the grant arrangement and the grant will be received. Grant payments received related to research and development costs incurred prior to the approval of the qualifying program are recognized immediately upon approval of the program by the grantor. |
Revenue recognition | Revenue recognition The Company generates revenue from research collaboration and license agreements with customers. Goods and services in the agreements may include the grant of licenses for the use of the Company’s technology, the provision of services associated with the research and development of product candidates, manufacturing services, and participation on joint steering committees. Such agreements may provide for consideration to the Company in the form of upfront payments; funding or reimbursement of research and development services; reimbursement of certain costs; option exercise payments; payments due upon the achievement of preclinical, clinical, regulatory, and sales-based milestones; and royalty payments on licensed products. On January 1, 2018, the Company adopted the new revenue recognition standard, discussed below under the heading “Recent accounting pronouncements,” which amended revenue recognition principles and provides a single, comprehensive set of criteria for revenue recognition. The new revenue standard applies to all contracts with customers except for contracts that are within the scope of other standards. The new guidance provides a five-step framework through which revenue is recognized when control of promised goods or services is transferred to a customer at an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. To determine revenue recognition for arrangements that the Company concludes are within the scope of the new revenue recognition standard, management performs the following five steps: (i) identifies the contract(s) with a customer; (ii) identifies the performance obligations in the contract; (iii) determines the transaction price, including whether there are any constraints on variable consideration; (iv) allocates the transaction price to the performance obligations; and (v) recognizes revenue when (or as) the Company satisfies a performance obligation. At contract inception, once a contract is determined to be within the scope of the new revenue standard, Dicerna assesses whether individual goods or services promised within each contract are distinct and, therefore, represent a separate performance obligation. Goods and services that are determined not to be distinct are combined with other promised goods and services until a distinct bundle is identified. Dicerna allocates the transaction price (the amount of consideration to which the Company expects to be entitled in exchange for the promised goods or services) to each performance obligation and recognizes the associated revenue when (or as) each performance obligation is satisfied. The Company’s estimate of the transaction price for each contract includes all variable consideration to which Dicerna expects to be entitled at each measuring period. When two or more contracts are entered into with the same customer at or near the same time, the Company evaluates the contracts to determine whether the contracts should be accounted for as a single arrangement. Contracts are combined and accounted for as a single arrangement if one or more of the following criteria are met: (i) the contracts are negotiated as a package with a single commercial objective; (ii) the amount of consideration to be paid in one contract depends on the price or performance of the other contract; or (iii) the goods or services promised in the contracts (or some goods or services promised in each of the contracts) are a single performance obligation. The evaluation of whether promised goods or services represent distinct performance obligations is subjective and requires the Company to make judgments about the promised goods and services and whether such goods and services are separable from the other aspects of the contract(s). The transaction price is allocated among the performance obligations on a relative standalone selling price basis, and the applicable revenue recognition criteria are applied to each of the separate performance obligations. The Company may estimate the standalone selling price using a residual method when the selling price is highly variable because a representative standalone selling price is not discernible from past transactions or other observable evidence, or when the selling price is uncertain. Determining the standalone selling price for performance obligations requires significant judgment. When an observable price of a promised good or service is not readily available, the Company considers relevant assumptions to estimate the standalone selling price, including, as applicable, market conditions, development timelines, probabilities of technical and regulatory success, reimbursement rates for personnel costs, forecasted revenues, potential limitations to the selling price of the product, and discount rates. The Company applies judgment in determining whether a combined performance obligation is satisfied at a point in time or over time, and, if over time, concluding upon the appropriate method of measuring progress to be applied for purposes of recognizing revenue. The Company evaluates the measure of progress each reporting period and, as estimates related to the measure of progress change, related revenue recognition is adjusted accordingly. Changes in the Company’s estimated measure of progress are accounted for prospectively as a change in accounting estimate. The Company receives payments from its licensees as established in each contract. Upfront payments and fees are recorded as deferred revenue upon receipt or when due and most often require deferral of revenue recognition to a future period until the Company performs its obligations under the underlying arrangements. Where applicable, amounts are recorded as contracts receivable when the Company’s right to consideration is unconditional. Licenses of intellectual property: If a license granted to a customer to use the Company’s intellectual property is determined to be distinct from the other performance obligations identified in the arrangement, the Company recognizes revenue from consideration allocated to the license when the license is transferred to the licensee and the licensee is able to use and benefit from the license. For licenses that are bundled with other promises, the Company applies judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, to conclude upon the appropriate method of measuring progress for purposes of recognizing revenue related to consideration allocated to the performance obligation. Research and development services: Arrangements that include a promise for the Company to provide research or development services are assessed to determine whether the services are capable of being distinct, are not highly interdependent or do not significantly modify one another, and if so, the services are accounted for as a separate performance obligation as the services are provided to the customer. Otherwise, when research or development services are determined not to be capable of being distinct, such services are added to the performance obligation that includes the underlying license. For research and development services that are bundled with other promises, the Company applies judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, to conclude upon the appropriate method of measuring progress for purposes of recognizing revenue related to consideration allocated to the performance obligation. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition. Options: Customer options, such as options granted to allow a licensee to choose to research and develop additional or reserve product candidates against target genes to be identified in the future, or options that allow a customer to designate a target as a lead product, are evaluated at contract inception in order to determine whether those options provide a material right (i.e., an optional good or service offered for free or at a discount) to the customer. If the customer options represent a material right, the material right is treated as a separate performance obligation at the outset of the arrangement. The Company allocates the transaction price to material rights based on the standalone selling price, and revenue is recognized when or as the future goods or services are transferred or when the option expires. Customer options that are not material rights do not give rise to a separate performance obligation, and as such, the additional consideration that would result from a customer exercising an option in the future is not included in the transaction price for the current contract. Instead, the option is deemed a marketing offer, and additional option fee payments are recognized or begin being recognized as revenue when the licensee exercises the option. The exercise of an option that does not represent a material right is treated as a separate contract for accounting purposes. Milestone payments: At the inception of each contract with a customer that includes development or regulatory milestone payments, the Company evaluates whether the milestones are considered probable of being reached and estimates the amount to be included in the transaction price using the most likely amount method. If the Company concludes it is probable that a significant revenue reversal would not occur, the associated milestone payment is included in the transaction price. Milestone payments that are not within the control of the Company or the licensee, such as regulatory approvals, are generally not considered probable of being achieved until those approvals are received. The transaction price is then allocated to each performance obligation on a relative stand-alone selling price basis, for which the Company recognizes revenue as or when the performance obligations under the contract are satisfied. At the end of each subsequent reporting period, the Company re-evaluates the probability of achievement of all milestones and any related constraints, and, if necessary, adjusts the estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis and are recorded as revenue and through earnings in the period of adjustment. Royalties: For arrangements that include sales-based royalties, including milestone payments based on the level of sales, and when the license is deemed to be the predominant item to which the royalties relate, the Company recognizes revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). Contract costs: The Company recognizes as an asset the incremental costs of obtaining a contract with a customer if the costs are expected to be recovered. The Company has elected a practical expedient wherein it recognizes the incremental costs of obtaining a contract as an expense when incurred if the amortization period of the asset that it otherwise would have recognized is one year or less. To date, the Company has not incurred any incremental costs of obtaining a contract with a customer. Contract modifications: Contract modifications, defined as changes in the scope or price (or both) of a contract that are approved by the parties to the contract, such as a contract amendment, exist when the parties to a contract approve a modification that either creates new or changes existing enforceable rights and obligations of the parties to the contract. Depending on facts and circumstances, the Company accounts for a contract modification as one of the following: (i) a separate contract; (ii) a termination of the existing contract and a creation of a new contract; or (iii) a combination of the preceding treatments. A contract modification is accounted for as a separate contract if the scope of the contract increases because of the addition of promised goods or services that are distinct and the price of the contract increases by an amount of consideration that reflects the Company’s standalone selling prices of the additional promised goods or services. When a contract modification is not considered a separate contract and the remaining goods or services are distinct from the goods or services transferred on or before the date of the contract modification, the Company accounts for the contract modification as a termination of the existing contract and a creation of a new contract. When a contract modification is not considered a separate contract and the remaining goods or services are not distinct, the Company accounts for the contract modification as an add-on to the existing contract and as an adjustment to revenue on a cumulative catch-up basis. The Company receives payments from its licensees as established in each contract. Upfront payments and fees are recorded as deferred revenue upon receipt or when due and may require deferral of revenue recognition to a future period until the Company performs its obligations under these arrangements. Where applicable, amounts are recorded as contracts receivable when the Company’s right to consideration is unconditional. The Company does not assess whether a contract with a customer has a significant financing component if the expectation at contract inception is such that the period between payment by the licensees and the transfer of the promised goods or services to the licensees will be one year or less. |
Stock-based compensation | Stock-based compensation The Company’s stock-based compensation cost is measured at the grant date of the stock-based award based on the fair value of the award and is recognized as expense over the requisite service period, which generally represents the vesting period, and includes an estimate of the awards that will be forfeited. The Company uses the Black-Scholes valuation model for estimating the fair value of stock options. The fair value of stock option awards is affected by the valuation assumptions, including the expected volatility based on comparable market participants, expected term of the option, risk-free interest rate and expected dividends. |
Income taxes | Income taxes The Company records deferred tax assets and liabilities for the expected future tax consequences of temporary differences between the Company’s financial statement carrying amounts and the tax basis of assets and liabilities using enacted tax rates expected to be in effect in the years in which the differences are expected to reverse. A valuation allowance is provided to reduce the net deferred tax assets to the amount that will more likely than not be realized. The Company also assesses the probability that the positions taken or expected to be taken in its income tax returns will be sustained by taxing authorities. A “more likely than not” (more than 50 percent ) recognition threshold must be met before a tax benefit can be recognized. Tax positions that are more likely than not to be sustained are reflected in the Company’s consolidated financial statements. Tax positions are measured as the largest amount of tax benefit that is greater than 50 percent likely of being realized upon settlement with a taxing authority that has full knowledge of all relevant information. The difference between the benefit recognized for a position and the tax benefit claimed on a tax return is referred to as an unrecognized tax benefit. Potential interest and penalties associated with such uncertain tax positions are recorded as a component of income tax expense. |
Net loss per common share attributable to common stockholders | Net loss per common share attributable to common stockholders The Company computes basic net loss per common share by dividing net loss attributable to common stockholders by the weighted‑average number of common shares outstanding. In periods of net income, the Company’s accounting policy includes allocating a proportional share of net income to participating securities, as determined by dividing total weighted‑average participating securities by the sum of the total weighted‑average common shares and participating securities (the “two-class method”). The Company’s nonvested restricted shares participated in any dividends declared by the Company and were therefore considered to be participating securities. Participating securities have the effect of diluting both basic and diluted earnings per share during periods of income. During periods when the Company incurred a net loss, the Company did not allocate a loss to participating securities because they had no contractual obligation to share in the losses of the Company. The Company computes diluted net loss per common share after giving consideration to the dilutive effect of stock options, warrants, nonvested restricted stock, and redeemable convertible preferred shares that are outstanding during the period, except where such non-participating securities would be anti-dilutive. The outstanding securities presented below were excluded from the calculation of net loss per share attributable to common stockholders because such securities would have been anti-dilutive due to the Company’s net loss per share attributable to common stockholders during the periods ending on the dates presented. DECEMBER 31, 2018 2017 2016 Options to purchase common stock 7,787,690 6,124,096 5,099,449 Warrants to purchase common stock 2,198 87,901 87,901 Nonvested restricted common stock — 10,000 20,000 Total 7,789,888 6,221,997 5,207,350 |
Comprehensive loss | Comprehensive loss Comprehensive loss is defined as the change in stockholders’ equity of a business enterprise during a period from transactions and other events and circumstances from non-owner sources. The Company has no comprehensive loss items other than net loss. |
Recent accounting pronouncements and change in accounting principle | Recent accounting pronouncements The following table provides a description of the recent accounting pronouncements that may have a material effect on the Company’s consolidated financial statements: Standard Description Effective Date Effect on the Consolidated Accounting Standards Adopted During the Year Ended December 31, 2018 ASU 2014-09, Revenue from Contracts with Customers (Topic 606) and related amendments (“ASC 606”) This ASU amends the guidance for accounting for revenue from contracts with customers, superseding the revenue recognition requirements in ASC 605, Revenue Recognition . ASC 606 was effective for annual reporting periods beginning after December 15, 2017. Under ASC 606, two adoption methods were allowed: retrospectively to all prior reporting periods presented, with certain practical expedients permitted, or retrospectively with the cumulative effect of initially adopting ASC 606 recognized at the date of initial application. January 1, 2018 Effective January 1, 2018, the Company adopted the requirements of ASC 606 using the full retrospective method, which required the Company to recast the prior reporting periods presented. All financial statements and disclosures have been recast to comply with ASC 606. See “Change in accounting principle” below for a summary of the amounts by which each financial statement line item was affected by the adoption of ASC 606. – Collaborative Research and License Agreements ). ASU No. 2016-18, Statement of Cash Flows (Topic 230): Restricted Cash (“ASU 2016-18”) In November 2016, the FASB issued ASU No. 2016-18, Statement of Cash Flows (Topic 230): Restricted Cash (“ASU 2016-18”), a consensus of the FASB’s EITF. ASU 2016-18 requires that the statement of cash flows explain the change during the period in the total of cash and cash equivalents, including amounts generally described as restricted cash or restricted cash equivalents. Entities are required to reconcile such total to amounts on the balance sheet and disclose the nature of the restrictions. By requiring that the statement of cash flows explain the change during the period in the total of cash, cash equivalents and restricted cash, the new guidance eliminates current diversity in practice. January 1, 2018 The Company adopted ASU 2016-18 on January 1, 2018 and applied this new guidance retrospectively to all periods presented. Consequently, transfers between restricted and unrestricted cash equivalents accounts are no longer reported as a cash flow in the Company’s consolidated statement of cash flows. As a result of the adoption of this standard, the Company includes its restricted cash equivalents balance in the cash and cash equivalents reconciliation of operating, investing and financing activities. The retrospective adoption resulted in the inclusion of restricted cash equivalents of $0.7 million, and $1.1 million in the consolidated statements of cash flows as of December 31, 2017 and 2016, respectively. Standard Description Effective Date for Company Effect on the Consolidated Financial Statements Recently Issued Accounting Standards Not Yet Adopted ASU 2016-02, Leases (Topic 842) This ASU supersedes existing accounting standards for lease accounting, including requiring lessees to recognize most leases on their balance sheets and making targeted changes to lessor accounting. ASU 2016-02 will be effective for the Company beginning in the first quarter of 2019. ASU 2016-02 requires a modified retrospective transition approach for all leases existing at, or entered into after, the date of initial application, with an option to use certain transition relief. In July 2018, the FASB issued ASU 2018-11, Leases (Topic 842): Targeted Improvements (“ASU 2018-11”), which allows entities to initially apply the new lease guidance at the adoption date and recognize a cumulative-effect adjustment to the opening balance of retained earnings in the period of adoption. January 1, 2019 Management expects that the adoption of ASU 2016-02 will result in the recognition of a right of use asset and related liability associated with the Company’s non-cancelable operating lease arrangements for office and laboratory spaces (see Note 14 – Commitments and Contingencies and Note 17 - Subsequent Events ). The Company is in the process of determining whether it will utilize the optional transition method presented in ASU 2018-11. Change in accounting principle In May 2014, the Financial Accounting Standards Board ("FASB") issued ASC 606. Under the standard, revenue is recognized when a customer obtains control of promised goods or services in an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. Effective January 1, 2018, the Company adopted ASC 606 using the full retrospective method, which required the Company to recast the prior reporting periods presented. The Company has recast its consolidated financial statements from amounts previously reported due to the adoption of ASC 606. Select Consolidated Statement of Operations line items, which reflect the impact of the adoption of ASC 606, are as follows: YEAR ENDED DECEMBER 31, 2017 AS REPORTED ADJUSTMENTS AS ADJUSTED Revenue from collaborative arrangements $ 1,182 $ (152 ) $ 1,030 Loss from operations $ (60,587 ) $ (152 ) $ (60,739 ) Net loss $ (60,048 ) $ (152 ) $ (60,200 ) Net loss attributable to common stockholders $ (80,140 ) $ (152 ) $ (80,292 ) The adoption of ASC 606 did not have an impact on net loss per share attributable to common stockholders for any period presented. Select Consolidated Balance Sheet line items, which reflect the adoption of ASC 606, are as follows: DECEMBER 31, 2017 AS REPORTED ADJUSTMENTS AS ADJUSTED Prepaid expenses and other current assets $ 3,297 $ 118 $ 3,415 Current portion of deferred revenue $ 6,000 $ 180 $ 6,180 Deferred revenue, net of current portion $ 3,000 $ 90 $ 3,090 Accumulated deficit $ (315,804 ) $ (152 ) $ (315,956 ) The adoption of ASC 606 did not have an impact on net cash used in operating, investing, or financing activities in the Company’s Consolidated Statements of Cash Flows. |
Held-to-maturity investments | The Company invests its excess cash balances in short-term and long-term fixed-income investments. The Company determines the appropriate classification of investments at the time of purchase and re-evaluates such designation as of each balance sheet date. Debt securities carried at amortized cost are classified as held-to-maturity when the Company has the positive intent and ability to hold the securities to maturity. The Company’s investment policy mandates that, at the time of purchase, the maturity of each investment within its portfolio shall not exceed 24 months . In addition, the weighted‑average maturity of the investment portfolio must not exceed 12 months . |
Fair value measurements | Fair value is an exit price, representing the amount that would be received from the sale of an asset or paid to transfer a liability in an orderly transaction between market participants. As such, fair value is a market-based measurement that should be determined based on assumptions that market participants would use in pricing an asset or liability. Valuation techniques used to measure fair value are performed in a manner to maximize the use of observable inputs and minimize the use of unobservable inputs. As a basis for considering such assumption the accounting literature establishes a three-tier value hierarchy which prioritizes the inputs used in measuring fair value as follows: • Level 1 – observable inputs, such as quoted prices in active markets; • Level 2 – inputs other than the quoted prices in active markets that are observable either directly or indirectly; and • Level 3 – unobservable inputs for which there is little or no market data, which requires the Company to develop its own assumptions. |
Legal proceedings | Legal proceedings From time to time, the Company may be subject to various claims and legal proceedings. If the potential loss from any claim, asserted or unasserted, or legal proceeding is considered probable and the amount is reasonably estimable, the Company will accrue a liability for the estimated loss. |
SUMMARY OF SIGNIFICANT ACCOUN_3
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Accounting Policies [Abstract] | |
Estimated Useful Lives | Depreciation is provided using the straight-line method over the estimated useful lives, as shown below: ASSET CATEGORY ESTIMATED Laboratory equipment 5 years Office and computer equipment 3 - 5 years Furniture and fixtures 5 years Leasehold improvements 5 years or the remaining |
Schedule of Antidilutive Securities Excluded from Computation of Earnings Per Share | The outstanding securities presented below were excluded from the calculation of net loss per share attributable to common stockholders because such securities would have been anti-dilutive due to the Company’s net loss per share attributable to common stockholders during the periods ending on the dates presented. DECEMBER 31, 2018 2017 2016 Options to purchase common stock 7,787,690 6,124,096 5,099,449 Warrants to purchase common stock 2,198 87,901 87,901 Nonvested restricted common stock — 10,000 20,000 Total 7,789,888 6,221,997 5,207,350 |
Schedule of Recent Accounting Pronouncements | Recent accounting pronouncements The following table provides a description of the recent accounting pronouncements that may have a material effect on the Company’s consolidated financial statements: Standard Description Effective Date Effect on the Consolidated Accounting Standards Adopted During the Year Ended December 31, 2018 ASU 2014-09, Revenue from Contracts with Customers (Topic 606) and related amendments (“ASC 606”) This ASU amends the guidance for accounting for revenue from contracts with customers, superseding the revenue recognition requirements in ASC 605, Revenue Recognition . ASC 606 was effective for annual reporting periods beginning after December 15, 2017. Under ASC 606, two adoption methods were allowed: retrospectively to all prior reporting periods presented, with certain practical expedients permitted, or retrospectively with the cumulative effect of initially adopting ASC 606 recognized at the date of initial application. January 1, 2018 Effective January 1, 2018, the Company adopted the requirements of ASC 606 using the full retrospective method, which required the Company to recast the prior reporting periods presented. All financial statements and disclosures have been recast to comply with ASC 606. See “Change in accounting principle” below for a summary of the amounts by which each financial statement line item was affected by the adoption of ASC 606. – Collaborative Research and License Agreements ). ASU No. 2016-18, Statement of Cash Flows (Topic 230): Restricted Cash (“ASU 2016-18”) In November 2016, the FASB issued ASU No. 2016-18, Statement of Cash Flows (Topic 230): Restricted Cash (“ASU 2016-18”), a consensus of the FASB’s EITF. ASU 2016-18 requires that the statement of cash flows explain the change during the period in the total of cash and cash equivalents, including amounts generally described as restricted cash or restricted cash equivalents. Entities are required to reconcile such total to amounts on the balance sheet and disclose the nature of the restrictions. By requiring that the statement of cash flows explain the change during the period in the total of cash, cash equivalents and restricted cash, the new guidance eliminates current diversity in practice. January 1, 2018 The Company adopted ASU 2016-18 on January 1, 2018 and applied this new guidance retrospectively to all periods presented. Consequently, transfers between restricted and unrestricted cash equivalents accounts are no longer reported as a cash flow in the Company’s consolidated statement of cash flows. As a result of the adoption of this standard, the Company includes its restricted cash equivalents balance in the cash and cash equivalents reconciliation of operating, investing and financing activities. The retrospective adoption resulted in the inclusion of restricted cash equivalents of $0.7 million, and $1.1 million in the consolidated statements of cash flows as of December 31, 2017 and 2016, respectively. Standard Description Effective Date for Company Effect on the Consolidated Financial Statements Recently Issued Accounting Standards Not Yet Adopted ASU 2016-02, Leases (Topic 842) This ASU supersedes existing accounting standards for lease accounting, including requiring lessees to recognize most leases on their balance sheets and making targeted changes to lessor accounting. ASU 2016-02 will be effective for the Company beginning in the first quarter of 2019. ASU 2016-02 requires a modified retrospective transition approach for all leases existing at, or entered into after, the date of initial application, with an option to use certain transition relief. In July 2018, the FASB issued ASU 2018-11, Leases (Topic 842): Targeted Improvements (“ASU 2018-11”), which allows entities to initially apply the new lease guidance at the adoption date and recognize a cumulative-effect adjustment to the opening balance of retained earnings in the period of adoption. January 1, 2019 Management expects that the adoption of ASU 2016-02 will result in the recognition of a right of use asset and related liability associated with the Company’s non-cancelable operating lease arrangements for office and laboratory spaces (see Note 14 – Commitments and Contingencies and Note 17 - Subsequent Events ). The Company is in the process of determining whether it will utilize the optional transition method presented in ASU 2018-11. The Company has recast its consolidated financial statements from amounts previously reported due to the adoption of ASC 606. Select Consolidated Statement of Operations line items, which reflect the impact of the adoption of ASC 606, are as follows: YEAR ENDED DECEMBER 31, 2017 AS REPORTED ADJUSTMENTS AS ADJUSTED Revenue from collaborative arrangements $ 1,182 $ (152 ) $ 1,030 Loss from operations $ (60,587 ) $ (152 ) $ (60,739 ) Net loss $ (60,048 ) $ (152 ) $ (60,200 ) Net loss attributable to common stockholders $ (80,140 ) $ (152 ) $ (80,292 ) The adoption of ASC 606 did not have an impact on net loss per share attributable to common stockholders for any period presented. Select Consolidated Balance Sheet line items, which reflect the adoption of ASC 606, are as follows: DECEMBER 31, 2017 AS REPORTED ADJUSTMENTS AS ADJUSTED Prepaid expenses and other current assets $ 3,297 $ 118 $ 3,415 Current portion of deferred revenue $ 6,000 $ 180 $ 6,180 Deferred revenue, net of current portion $ 3,000 $ 90 $ 3,090 Accumulated deficit $ (315,804 ) $ (152 ) $ (315,956 ) |
HELD-TO-MATURITY INVESTMENTS (T
HELD-TO-MATURITY INVESTMENTS (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Investments, Debt and Equity Securities [Abstract] | |
Schedule of Held-To-Maturity Investments | The following tables provide information relating to the Company’s held-to-maturity investments: DECEMBER 31, 2018 DESCRIPTION AMORTIZED COST GROSS UNREALIZED GAINS GROSS LOSSES FAIR VALUE U.S. Treasury securities maturing in one year or less $ 248,387 $ — $ (43 ) $ 248,344 DECEMBER 31, 2017 DESCRIPTION AMORTIZED COST GROSS UNREALIZED GAINS GROSS LOSSES FAIR VALUE U.S. Treasury securities maturing in one year or less $ 44,889 $ — $ (30 ) $ 44,859 |
FAIR VALUE MEASUREMENTS (Tables
FAIR VALUE MEASUREMENTS (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Fair Value Disclosures [Abstract] | |
Schedule of Assets Measured or Disclosed at Fair Value | A summary of the Company’s assets that are measured or disclosed at fair value on a recurring basis is presented below: DECEMBER 31, 2018 DESCRIPTION TOTAL FAIR VALUE LEVEL 1 LEVEL 2 LEVEL 3 Cash equivalents Money market funds $ 44,886 $ 44,886 $ — $ — Held-to-maturity investment U.S. Treasury securities 248,344 — 248,344 — Restricted cash equivalents Money market funds 744 — 744 — Total $ 293,974 $ 44,886 $ 249,088 $ — DECEMBER 31, 2017 DESCRIPTION TOTAL FAIR VALUE LEVEL 1 LEVEL 2 LEVEL 3 Cash equivalents Money market funds $ 51,441 $ 51,441 $ — $ — Held-to-maturity investments U.S. Treasury securities 44,859 — 44,859 — Restricted cash equivalents Money market funds 744 — 744 — Total $ 97,044 $ 51,441 $ 45,603 $ — |
PREPAID EXPENSES AND OTHER CU_2
PREPAID EXPENSES AND OTHER CURRENT ASSETS (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract] | |
Summary of Prepaid Expenses and Other Current Assets | Prepaid expenses and other current assets consist of the following: DECEMBER 31, 2018 2017 Prepaid clinical, contract research, and manufacturing costs $ 1,419 $ 1,931 Interest receivable and other current assets 815 391 Prepaid insurance 341 318 Prepaid rent 245 239 Other 68 536 Prepaid expenses and other current assets $ 2,888 $ 3,415 |
PROPERTY AND EQUIPMENT, NET (Ta
PROPERTY AND EQUIPMENT, NET (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Property, Plant and Equipment [Abstract] | |
Summary of Property and Equipment, Net | Property and equipment, net, consists of the following : DECEMBER 31, 2018 2017 Laboratory equipment $ 4,607 $ 4,410 Office and computer equipment 1,021 900 Furniture and fixtures 479 479 Leasehold improvements 257 257 Construction in process 1,661 — Property and equipment, at cost 8,025 6,046 Less accumulated depreciation and amortization (5,307 ) (4,534 ) Property and equipment, net $ 2,718 $ 1,512 |
ACCRUED EXPENSES AND OTHER CU_2
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses and Other Current Liabilities | Accrued expenses and other current liabilities consist of the following: DECEMBER 31, 2018 2017 Accrued clinical, contract research, and manufacturing costs $ 3,960 $ 1,860 Accrued compensation and related benefits 3,684 1,987 Accrued professional fees 1,693 1,488 Accrued other expenses 312 391 Accrued expenses and other current liabilities $ 9,649 $ 5,726 |
COLLABORATIVE RESEARCH AND LI_2
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Revenue from Contract with Customer [Abstract] | |
Contract with Customer, Asset and Liability | The following table presents changes in the Company’s aggregate deferred revenue balances for each reporting period: YEAR ENDED BALANCE AT BEGINNING OF PERIOD ADDITIONS DEDUCTIONS BALANCE AT END OF PERIOD Deferred revenue, current and noncurrent $ 9,270 $ 180,092 $ (6,176 ) $ 183,186 YEAR ENDED BALANCE AT BEGINNING OF PERIOD ADDITIONS DEDUCTIONS BALANCE AT END OF PERIOD Deferred revenue, current and noncurrent $ — $ 10,300 $ (1,030 ) $ 9,270 |
REDEEMABLE CONVERTIBLE PREFER_2
REDEEMABLE CONVERTIBLE PREFERRED STOCK (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Summary of Changes in Redeemable Convertible Preferred | The following table reflects the changes in rede emable convertible preferred shares recorded during the year ended December 31, 2017: Balance at January 1, 2017 $ — Issuance of redeemable convertible preferred shares 70,000 Share issuance costs (750 ) Net proceeds 69,250 Discount resulting from the BCF at issuance (6,144 ) Accretion of the discount resulting from the BCF (deemed dividend) 6,144 Dividends accrued at the stated rates 5,515 Fair value in excess of dividends accrued at the stated rates 3,846 Accretion of share issuance costs (additional dividends) 750 Balance immediately prior to conversion 79,361 Conversion of redeemable convertible preferred shares (79,361 ) Balance at December 31, 2017 $ — |
REDEEMABLE CONVERTIBLE PREFERRED STOCK | |
Schedule of Valuation Assumptions | The lattice model was used to determine fair value of dividends on each dividend date through September 30, 2017, which was the last dividend date prior to conversion of the redeemable convertible preferred shares, included the following inputs: JUNE 30, SEPTEMBER 30, Price per common share $ 3.17 $ 5.75 Expected term (in years) 6.75 6.50 Expected volatility 70.0 % 73.0 % Risk-adjusted discount rate 18.0 % 19.1 % |
STOCK-BASED COMPENSATION (Table
STOCK-BASED COMPENSATION (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Stock-Based Compensation Expense | The Company has classified stock-based compensation expense in its consolidated statements of operations as follows: YEAR ENDED DECEMBER 31, 2018 2017 2016 Research and development expenses $ 3,062 $ 3,536 $ 4,467 General and administrative expenses 4,826 4,234 4,698 Total $ 7,888 $ 7,770 $ 9,165 |
Stock Option Activity for Employee and Nonemployee | The table below summarizes the activity under the Company’s equity incentive plans: NUMBER WEIGHTED- EXERCISE WEIGHTED- AGGREGATE INTRINSIC VALUE OUTSTANDING – JANUARY 1, 2018 6,124,096 $ 8.58 Granted 2,241,350 $ 11.07 Exercised (329,934 ) $ 5.31 Forfeited/Canceled (90,026 ) $ 5.71 Expired (157,796 ) $ 14.08 OUTSTANDING – DECEMBER 31, 2018 7,787,690 $ 9.36 7.3 $ 24,305 EXERCISABLE – DECEMBER 31, 2018 4,842,084 $ 9.71 6.4 $ 16,151 VESTED AND EXPECTED TO VEST – DECEMBER 31, 2018 7,567,801 $ 9.30 7.2 $ 23,948 |
Stock Options Granted to Non-Employees | |
Schedule of Valuation Assumptions | The assumptions used in the Black-Scholes option-pricing model for all stock options granted during each period presented are as follows: YEAR ENDED DECEMBER 31, 2018 2017 2016 Common stock price $9.14 - $15.74 $2.49 – $9.71 $2.94 – $9.09 Expected option term (in years) 5.50 - 6.25 5.50 – 6.25 5.50 – 6.25 Expected volatility 75.9% - 78.3% 79.4% – 91.1% 70.9% – 79.4% Risk-free interest rate 2.3% - 3.0% 1.9% – 2.2% 1.2% – 2.0% Expected dividend yield 0.0% 0.0% 0.0% |
INCOME TAXES (Tables)
INCOME TAXES (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Income Tax Disclosure [Abstract] | |
Reconciliation Between Income Taxes Computed at the Federal Statutory Income Tax Rate and the Provision | The reconciliation between income taxes computed at the federal statutory income tax rate and the provision for (benefit from) income taxes is as follows: YEAR ENDED DECEMBER 31, 2018 2017 2016 Federal statutory rate 21.0 % 34.0 % 34.0 % Effect of: Foreign rate differential (9.5 )% (17.6 )% (31.4 )% Tax reform — % (29.6 )% — % Net operating loss limitation (23.0 )% — % — % Change in valuation allowance 10.6 % 13.5 % — % Research and development tax credit 0.5 % 0.6 % (0.7 )% Stock-based compensation expense 0.4 % (0.8 )% (0.9 )% Other — % (0.1 )% (1.0 )% Total — % — % — % |
Deferred Tax Assets | The components of the Company’s deferred tax assets are as follows: DECEMBER 31, 2018 2017 Deferred tax assets: Net operating loss carryforwards $ 24,739 $ 32,008 Capitalized research and development costs 516 618 Research and development credit carryforwards 3,988 3,481 Stock-based compensation expense 7,644 6,066 Depreciation expense and other costs 75 42 Net deferred tax assets 36,962 42,215 Valuation allowance (36,962 ) (42,215 ) Net deferred tax assets $ — $ — |
Unrecognized Tax Benefit | A reconciliation of the gross unrecognized tax benefit is as follows: YEAR ENDED DECEMBER 31, 2018 2017 Unrecognized tax benefits at the beginning of the period $ 1,451 $ 1,210 Additions for current tax positions 211 243 Changes for previous tax positions (31 ) (2 ) Unrecognized tax benefits at the end of the period $ 1,631 $ 1,451 |
COMMITMENTS AND CONTINGENCIES (
COMMITMENTS AND CONTINGENCIES (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Commitments and Contingencies Disclosure [Abstract] | |
Summary of Future Minimum Lease Payments Payable | Future minimum lease payments on the Company’s non-cancelable operating lease for office and laboratory space are as follows: YEARS ENDING DECEMBER 31, OPERATING LEASE 2019 $ 1,678 2020 1,581 Total $ 3,259 |
QUARTERLY FINANCIAL DATA (UNA_2
QUARTERLY FINANCIAL DATA (UNAUDITED) (Tables) | 12 Months Ended |
Dec. 31, 2018 | |
Quarterly Financial Information Disclosure [Abstract] | |
Schedule of Quarterly Financial Data | The following tables contain selected quarterly financial information for the years ended December 31, 2018 and 2017 . The Company believes that the following information reflects all normal recurring adjustments necessary for a fair statement of the information for the periods presented. 2018 FIRST QUARTER SECOND QUARTER THIRD QUARTER FOURTH QUARTER TOTAL YEAR Revenue from collaborative arrangements $ 1,545 $ 1,545 $ 1,545 $ 1,541 $ 6,176 Net loss $ (15,579 ) $ (35,644 ) $ (19,020 ) $ (18,610 ) $ (88,853 ) Net loss attributable to common stockholders $ (15,579 ) $ (35,644 ) $ (19,020 ) $ (18,610 ) $ (88,853 ) Net loss per share attributable to common stockholders – basic and diluted $ (0.30 ) $ (0.68 ) $ (0.35 ) $ (0.29 ) $ (1.60 ) 2017 FIRST QUARTER SECOND QUARTER THIRD QUARTER FOURTH QUARTER TOTAL YEAR Revenue from collaborative arrangements $ — $ — $ — $ 1,030 $ 1,030 Net loss $ (14,201 ) $ (15,225 ) $ (15,033 ) $ (15,741 ) $ (60,200 ) Net loss attributable to common stockholders $ (14,201 ) $ (23,991 ) $ (19,144 ) $ (22,956 ) $ (80,292 ) Net loss per share attributable to common stockholders – basic and diluted $ (0.68 ) $ (1.15 ) $ (0.92 ) $ (0.91 ) $ (3.66 ) |
DESCRIPTION OF BUSINESS AND B_2
DESCRIPTION OF BUSINESS AND BASIS OF PRESENTATION - Additional Information (Details) - USD ($) | 3 Months Ended | 12 Months Ended | |||||||
Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||
Reclassify grant income | $ (1,541,000) | $ (1,545,000) | $ (1,545,000) | $ (1,545,000) | $ (1,030,000) | $ 0 | $ (6,176,000) | $ (1,030,000) | $ 0 |
Litigation expense | (29,132,000) | (9,043,000) | (2,916,000) | ||||||
Cash, cash equivalents, and held-to-maturity investments | 302,600,000 | 302,600,000 | |||||||
Contract receivables | 100,000,000 | 0 | 100,000,000 | 0 | |||||
Litigation settlement payable | (10,500,000) | $ 0 | (10,500,000) | 0 | |||||
Option Payment | |||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||
Near-term receipts | $ 5,000,000 | $ 5,000,000 | |||||||
Grant | Prior Year Reclassifications To Conform To Current Year Presentation | |||||||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||||||
Reclassify grant income | $ 1,100,000 | $ (300,000) |
SUMMARY OF SIGNIFICANT ACCOUN_4
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Narrative (Details) | 3 Months Ended | 12 Months Ended | |||||||
Dec. 31, 2018USD ($) | Sep. 30, 2018USD ($) | Jun. 30, 2018USD ($) | Mar. 31, 2018USD ($) | Dec. 31, 2017USD ($) | Sep. 30, 2017USD ($) | Dec. 31, 2018USD ($)segment | Dec. 31, 2017USD ($) | Dec. 31, 2016USD ($) | |
Schedule Of Significant Accounting Policies [Line Items] | |||||||||
Lease expiration date | Nov. 30, 2020 | ||||||||
Contract receivables | $ 100,000,000 | $ 0 | $ 100,000,000 | $ 0 | |||||
Long-lived assets, impairment charge | $ 0 | 0 | $ 0 | ||||||
Number of operating segment | segment | 1 | ||||||||
Revenue from collaborative arrangements | 1,541,000 | $ 1,545,000 | $ 1,545,000 | $ 1,545,000 | 1,030,000 | $ 0 | $ 6,176,000 | 1,030,000 | 0 |
Restricted cash equivalents | $ 744,000 | 744,000 | $ 744,000 | 744,000 | 1,116,000 | ||||
Accounting Standards Update 2016-18 | |||||||||
Schedule Of Significant Accounting Policies [Line Items] | |||||||||
Restricted cash equivalents | $ 700,000 | $ 700,000 | $ 1,100,000 | ||||||
Total Purchases | Customer Concentration Risk | |||||||||
Schedule Of Significant Accounting Policies [Line Items] | |||||||||
Customer percentage | 10.90% | ||||||||
Money market funds | |||||||||
Schedule Of Significant Accounting Policies [Line Items] | |||||||||
Lease expiration date | Nov. 30, 2020 |
SUMMARY OF SIGNIFICANT ACCOUN_5
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Estimated Useful Lives (Details) | 12 Months Ended |
Dec. 31, 2018 | |
Laboratory equipment | |
Property, Plant and Equipment [Line Items] | |
ESTIMATED USEFUL LIVES | 5 years |
Office and computer equipment | Minimum | |
Property, Plant and Equipment [Line Items] | |
ESTIMATED USEFUL LIVES | 3 years |
Office and computer equipment | Maximum | |
Property, Plant and Equipment [Line Items] | |
ESTIMATED USEFUL LIVES | 5 years |
Furniture and fixtures | |
Property, Plant and Equipment [Line Items] | |
ESTIMATED USEFUL LIVES | 5 years |
Leasehold improvements | |
Property, Plant and Equipment [Line Items] | |
ESTIMATED USEFUL LIVES | 5 years |
SUMMARY OF SIGNIFICANT ACCOUN_6
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Schedule of Antidilutive Securities Excluded from Computation of Earnings Per Share (Details) - shares | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Anti-dilutive securities (in shares) | 7,789,888 | 6,221,997 | 5,207,350 |
Options to purchase common stock | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Anti-dilutive securities (in shares) | 7,787,690 | 6,124,096 | 5,099,449 |
Warrants to purchase common stock | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Anti-dilutive securities (in shares) | 2,198 | 87,901 | 87,901 |
Nonvested restricted common stock | |||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | |||
Anti-dilutive securities (in shares) | 0 | 10,000 | 20,000 |
SUMMARY OF SIGNIFICANT ACCOUN_7
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Consolidated Statements of Operations (Details) - USD ($) | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Revenue from collaborative arrangements | $ 1,541,000 | $ 1,545,000 | $ 1,545,000 | $ 1,545,000 | $ 1,030,000 | $ 0 | $ 6,176,000 | $ 1,030,000 | $ 0 | ||
Loss from operations | (90,352,000) | (60,739,000) | (59,748,000) | ||||||||
Net loss | (18,610,000) | (19,020,000) | (35,644,000) | (15,579,000) | (15,741,000) | (15,033,000) | $ (15,225,000) | $ (14,201,000) | (88,853,000) | (60,200,000) | (59,513,000) |
Net loss attributable to common stockholders | $ (18,610,000) | $ (19,020,000) | $ (35,644,000) | $ (15,579,000) | $ (22,956,000) | $ (19,144,000) | $ (23,991,000) | $ (14,201,000) | $ (88,853,000) | (80,292,000) | $ (59,513,000) |
AS REPORTED | |||||||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Revenue from collaborative arrangements | 1,182,000 | ||||||||||
Loss from operations | (60,587,000) | ||||||||||
Net loss | (60,048,000) | ||||||||||
Net loss attributable to common stockholders | (80,140,000) | ||||||||||
Accounting Standards Update 2014-09 | ADJUSTMENTS | |||||||||||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||||||||||
Revenue from collaborative arrangements | (152,000) | ||||||||||
Loss from operations | (152,000) | ||||||||||
Net loss | (152,000) | ||||||||||
Net loss attributable to common stockholders | $ (152,000) |
SUMMARY OF SIGNIFICANT ACCOUN_8
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Consolidated Balance Sheet (Details) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||
Prepaid expenses and other current assets | $ 2,888 | $ 3,415 |
Current portion of deferred revenue | 68,893 | 6,180 |
Deferred revenue, net of current portion | 114,293 | 3,090 |
Accumulated deficit | $ (404,809) | (315,956) |
AS REPORTED | ||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||
Prepaid expenses and other current assets | 3,297 | |
Current portion of deferred revenue | 6,000 | |
Deferred revenue, net of current portion | 3,000 | |
Accumulated deficit | (315,804) | |
Accounting Standards Update 2014-09 | ADJUSTMENTS | ||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||
Prepaid expenses and other current assets | 118 | |
Current portion of deferred revenue | 180 | |
Deferred revenue, net of current portion | 90 | |
Accumulated deficit | $ (152) |
HELD-TO-MATURITY INVESTMENTS -
HELD-TO-MATURITY INVESTMENTS - Narrative (Details) | 12 Months Ended |
Dec. 31, 2018 | |
Schedule of Held-to-maturity Securities [Line Items] | |
Held-to-maturity investments portfolio maximum period | 24 months |
Maximum | |
Schedule of Held-to-maturity Securities [Line Items] | |
Weighted average maturity investment portfolio period | 12 months |
HELD-TO-MATURITY INVESTMENTS _2
HELD-TO-MATURITY INVESTMENTS - Schedule of Held-To-Maturity Investments (Details) - U.S. Treasury securities - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Schedule of Held-to-maturity Securities [Line Items] | ||
AMORTIZED COST | $ 248,387 | $ 44,889 |
GROSS UNREALIZED HOLDING GAINS | 0 | 0 |
GROSS UNREALIZED HOLDING LOSSES | (43) | (30) |
FAIR VALUE | $ 248,344 | $ 44,859 |
FAIR VALUE MEASUREMENTS - Sched
FAIR VALUE MEASUREMENTS - Schedule of Assets Measured or Disclosed at Fair Value (Details) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Restricted cash equivalents | ||
Total | $ 293,974 | $ 97,044 |
U.S. Treasury securities | ||
Held-to-maturity investment | ||
U.S. Treasury securities | 248,344 | 44,859 |
Money market funds | ||
Cash equivalents | ||
Money market funds | 44,886 | 51,441 |
Restricted cash equivalents | ||
Money market funds | 744 | 744 |
LEVEL 1 | ||
Restricted cash equivalents | ||
Total | 44,886 | 51,441 |
LEVEL 1 | U.S. Treasury securities | ||
Held-to-maturity investment | ||
U.S. Treasury securities | 0 | 0 |
LEVEL 1 | Money market funds | ||
Cash equivalents | ||
Money market funds | 44,886 | 51,441 |
Restricted cash equivalents | ||
Money market funds | 0 | 0 |
LEVEL 2 | ||
Restricted cash equivalents | ||
Total | 249,088 | 45,603 |
LEVEL 2 | U.S. Treasury securities | ||
Held-to-maturity investment | ||
U.S. Treasury securities | 248,344 | 44,859 |
LEVEL 2 | Money market funds | ||
Cash equivalents | ||
Money market funds | 0 | 0 |
Restricted cash equivalents | ||
Money market funds | 744 | 744 |
LEVEL 3 | ||
Restricted cash equivalents | ||
Total | 0 | 0 |
LEVEL 3 | U.S. Treasury securities | ||
Held-to-maturity investment | ||
U.S. Treasury securities | 0 | 0 |
LEVEL 3 | Money market funds | ||
Cash equivalents | ||
Money market funds | 0 | 0 |
Restricted cash equivalents | ||
Money market funds | $ 0 | $ 0 |
FAIR VALUE MEASUREMENTS - Addit
FAIR VALUE MEASUREMENTS - Additional Information (Details) - USD ($) | Dec. 31, 2018 | Dec. 31, 2017 |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Estimated present value of cash obligation | $ 10,500,000 | $ 0 |
Litigation settlement, liability | $ 0 | |
Alnylam | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash obligation payable as part of litigation settlement | $ 10,500,000 |
PREPAID EXPENSES AND OTHER CU_3
PREPAID EXPENSES AND OTHER CURRENT ASSETS - Summary of Prepaid Expenses and Other Current Assets (Details) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract] | ||
Prepaid clinical, contract research, and manufacturing costs | $ 1,419 | $ 1,931 |
Interest receivable and other current assets | 815 | 391 |
Prepaid insurance | 341 | 318 |
Prepaid rent | 245 | 239 |
Other | 68 | 536 |
Prepaid expenses and other current assets | $ 2,888 | $ 3,415 |
PROPERTY AND EQUIPMENT, NET - S
PROPERTY AND EQUIPMENT, NET - Summary of Property and Equipment, Net (Details) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Property, Plant and Equipment [Line Items] | ||
Property and equipment, at cost | $ 8,025 | $ 6,046 |
Less accumulated depreciation and amortization | (5,307) | (4,534) |
Property and equipment, net | 2,718 | 1,512 |
Laboratory equipment | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, at cost | 4,607 | 4,410 |
Office and computer equipment | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, at cost | 1,021 | 900 |
Furniture and fixtures | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, at cost | 479 | 479 |
Leasehold improvements | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, at cost | 257 | 257 |
Construction in process | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, at cost | $ 1,661 | $ 0 |
PROPERTY AND EQUIPMENT, NET - A
PROPERTY AND EQUIPMENT, NET - Additional Information (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Property, Plant and Equipment [Abstract] | |||
Depreciation and amortization expense | $ 774 | $ 778 | $ 840 |
ACCRUED EXPENSES AND OTHER CU_3
ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES - Schedule of Accrued Expenses and Other Current Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Payables and Accruals [Abstract] | ||
Accrued compensation and related benefits | $ 3,960 | $ 1,860 |
Accrued clinical, contract research, and manufacturing costs | 3,684 | 1,987 |
Accrued professional fees | 1,693 | 1,488 |
Accrued other expenses | 312 | 391 |
Accrued expenses and other current liabilities | $ 9,649 | $ 5,726 |
COLLABORATIVE RESEARCH AND LI_3
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS - Lilly Collaboration and Share Purchase Agreements (Details) | Oct. 25, 2018USD ($)target$ / sharesshares | Dec. 31, 2018USD ($) | Sep. 30, 2018USD ($) | Jun. 30, 2018USD ($) | Mar. 31, 2018USD ($) | Dec. 31, 2017USD ($) | Sep. 30, 2017USD ($) | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($) | Dec. 31, 2016USD ($) |
Disaggregation of Revenue [Line Items] | ||||||||||
Revenue from collaborative arrangements | $ 1,541,000 | $ 1,545,000 | $ 1,545,000 | $ 1,545,000 | $ 1,030,000 | $ 0 | $ 6,176,000 | $ 1,030,000 | $ 0 | |
Deferred revenue | 183,186,000 | 9,270,000 | 183,186,000 | 9,270,000 | $ 0 | |||||
Current portion of deferred revenue | 68,893,000 | $ 6,180,000 | 68,893,000 | $ 6,180,000 | ||||||
Lilly Collaboration And License Agreement | ||||||||||
Disaggregation of Revenue [Line Items] | ||||||||||
Number of hepatocyte targets | target | 3 | |||||||||
Number of targets in excess to contemplate agreement | target | 10 | |||||||||
Initial non-refundable upfront payment | $ 100,000,000 | |||||||||
Payment due on first non-hepatocyte target achievement | $ 5,000,000 | |||||||||
Collaborative arrangement term | 10 years | |||||||||
Revenue from collaborative arrangements | 0 | |||||||||
Deferred revenue | 148,700,000 | 148,700,000 | ||||||||
Current portion of deferred revenue | $ 54,000,000 | $ 54,000,000 | ||||||||
Lilly Collaboration And License Agreement | Maximum | ||||||||||
Disaggregation of Revenue [Line Items] | ||||||||||
Development and commercialization milestones receivable | $ 350,000,000 | |||||||||
Lilly Share Issuance Agreement | ||||||||||
Disaggregation of Revenue [Line Items] | ||||||||||
Number of shares issuable (in shares) | shares | 5,414,185 | |||||||||
Sale of stock (in dollars per share) | $ / shares | $ 18.47 | |||||||||
Expected proceeds from issuance of common stock | $ 100,000,000 | |||||||||
Combined Agreements | ||||||||||
Disaggregation of Revenue [Line Items] | ||||||||||
Upfront compensation | 200,000,000 | |||||||||
Compensation recorded in equity upon the issuance of the shares | 51,300,000 | |||||||||
Consideration received | 148,700,000 | |||||||||
Variable consideration for potential development and regulatory milestone beyond the three initial | $ 0 |
COLLABORATIVE RESEARCH AND LI_4
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS - Alexion Collaboration and Equity Agreements (Details) | Oct. 22, 2018USD ($)targetcandidate$ / sharesshares | Dec. 31, 2018USD ($) | Sep. 30, 2018USD ($) | Jun. 30, 2018USD ($) | Mar. 31, 2018USD ($) | Dec. 31, 2017USD ($) | Sep. 30, 2017USD ($) | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($) | Dec. 31, 2016USD ($) |
Disaggregation of Revenue [Line Items] | ||||||||||
Revenue from collaborative arrangements | $ 1,541,000 | $ 1,545,000 | $ 1,545,000 | $ 1,545,000 | $ 1,030,000 | $ 0 | $ 6,176,000 | $ 1,030,000 | $ 0 | |
Deferred revenue, net of current portion | 114,293,000 | 3,090,000 | 114,293,000 | 3,090,000 | ||||||
Current portion of deferred revenue | 68,893,000 | $ 6,180,000 | 68,893,000 | $ 6,180,000 | ||||||
Alexion Collaborative Research And License Agreement | ||||||||||
Disaggregation of Revenue [Line Items] | ||||||||||
Number of candidates | candidate | 2 | |||||||||
Number of pathway targets | target | 2 | |||||||||
Initial non-refundable upfront payment | $ 22,000,000 | |||||||||
Collaborative arrangement option exercise fee for each of candidates selected | 10,000,000 | |||||||||
Aggregate sales milestones receivable | $ 160,000,000 | |||||||||
Collaborative arrangement term | 10 years | |||||||||
Compensation recorded in equity upon the issuance of the shares | $ 5,900,000 | |||||||||
Number of additional pathway targets | target | 2 | |||||||||
Transaction price | $ 37,400,000 | |||||||||
Contingent milestone payments | 9,500,000 | |||||||||
Potential developmental milestones receivable | 3 | |||||||||
Variable consideration for potential development and regulatory milestone beyond the three initial | 0 | |||||||||
Alexion Collaborative Research And License Agreement | Maximum | ||||||||||
Disaggregation of Revenue [Line Items] | ||||||||||
Additional payment receivable | 600,000,000 | |||||||||
Option exercise fee | 20,000,000 | |||||||||
Development milestones receivable for each product | $ 105,000,000 | |||||||||
Alexion Share Issuance Agreement | ||||||||||
Disaggregation of Revenue [Line Items] | ||||||||||
Number of shares issuable (in shares) | shares | 835,834 | |||||||||
Sale of stock (in dollars per share) | $ / shares | $ 17.95 | |||||||||
Expected proceeds from issuance of common stock | $ 15,000,000 | |||||||||
Alexion Agreements | ||||||||||
Disaggregation of Revenue [Line Items] | ||||||||||
Expected proceeds from issuance of common stock | 15,000,000 | |||||||||
Compensation recorded in equity upon the issuance of the shares | 9,100,000 | |||||||||
Transaction price | $ 5,900,000 | |||||||||
Revenue from collaborative arrangements | 100,000 | |||||||||
Deferred revenue, net of current portion | 31,300,000 | 31,300,000 | ||||||||
Current portion of deferred revenue | $ 11,700,000 | $ 11,700,000 |
COLLABORATIVE RESEARCH AND LI_5
COLLABORATIVE RESEARCH AND LICENSE AGREEMENTS - BI Agreement and Related Amendments (Details) - USD ($) | Oct. 27, 2017 | Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | Oct. 31, 2018 |
Disaggregation of Revenue [Line Items] | |||||||||||
Income taxes receivable | $ 0 | $ 1,583,000 | $ 0 | $ 1,583,000 | |||||||
Revenue from collaborative arrangements | 1,541,000 | $ 1,545,000 | $ 1,545,000 | $ 1,545,000 | 1,030,000 | $ 0 | 6,176,000 | 1,030,000 | $ 0 | ||
Current portion of deferred revenue | 68,893,000 | $ 6,180,000 | 68,893,000 | $ 6,180,000 | |||||||
Foreign Tax Authority | |||||||||||
Disaggregation of Revenue [Line Items] | |||||||||||
Income taxes receivable | $ 1,600,000 | ||||||||||
Boehringer Ingelheim Agreement | |||||||||||
Disaggregation of Revenue [Line Items] | |||||||||||
Initial non-refundable upfront payment | 10,000,000 | ||||||||||
Reimbursable third-party expenses | 300,000 | ||||||||||
Consideration receivable upon potential development and commercial milestones | 191,000,000 | ||||||||||
Potential developmental milestones receivable | 99,000,000 | ||||||||||
Net sales milestones | 95,000,000 | ||||||||||
Boehringer Ingelheim Agreement | Transferred over Time | |||||||||||
Disaggregation of Revenue [Line Items] | |||||||||||
Remaining performance obligation amount | 10,300,000 | ||||||||||
Additional Target Agreement | |||||||||||
Disaggregation of Revenue [Line Items] | |||||||||||
Initial non-refundable upfront payment | $ 5,000,000 | ||||||||||
Consideration receivable upon potential development and commercial milestones | 170,000,000 | 170,000,000 | |||||||||
Option exercise fee | $ 5,000,000 | 5,000,000 | |||||||||
Transaction price | $ 5,000,000 | ||||||||||
Collaborative arrangement term | 3 years | ||||||||||
Revenue from collaborative arrangements | $ 6,100,000 | ||||||||||
Current portion of deferred revenue | $ 3,200,000 | $ 3,200,000 |
COLLABORATIVE RESERACH AND LICE
COLLABORATIVE RESERACH AND LICENSE AGREEMENTS - Changes in Deferred Revenue (Details) - USD ($) | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Movement in Deferred Revenue [Roll Forward] | |||
BALANCE AT BEGINNING OF PERIOD | $ 3,090,000 | ||
ADDITIONS | 180,092,000 | $ 10,300,000 | |
DEDUCTIONS | (6,176,000) | (1,030,000) | |
BALANCE AT END OF PERIOD | 114,293,000 | 3,090,000 | |
Deferred revenue | $ 183,186,000 | $ 9,270,000 | $ 0 |
STOCKHOLDERS' EQUITY - Preferre
STOCKHOLDERS' EQUITY - Preferred Stock (Details) - $ / shares | Dec. 18, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Apr. 11, 2017 |
Equity [Abstract] | ||||
Preferred stock, shares authorized (in shares) | 5,000,000 | 5,000,000 | ||
Preferred stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 | ||
Preferred stock, shares outstanding (in shares) | 0 | 0 | ||
Redeemable convertible preferred stock, issued (in shares) | 700,000 | 700,000 | ||
Conversion of redeemable convertible preferred stock (in shares) | 24,206,663 |
STOCKHOLDERS' EQUITY - Issuance
STOCKHOLDERS' EQUITY - Issuances of Common Stock (Details) - USD ($) $ / shares in Units, $ in Thousands | Dec. 19, 2018 | Oct. 25, 2018 | Oct. 22, 2018 | Sep. 11, 2018 | Apr. 20, 2018 | Dec. 18, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 |
Subsidiary, Sale of Stock [Line Items] | |||||||||
Issuance of common stock (in shares) | 983,208 | ||||||||
Aggregate proceeds | $ 115,000 | $ 46,000 | $ 108,099 | $ 43,225 | $ 0 | ||||
Underwriting fees and issuance costs | $ 3,200 | $ 330 | $ 3,221 | ||||||
Alexion Share Issuance Agreement | |||||||||
Subsidiary, Sale of Stock [Line Items] | |||||||||
Number of shares issuable (in shares) | 835,834 | ||||||||
Price per share (in dollars per share) | $ 17.95 | ||||||||
Share purchase price allocated to equity | $ 9,100 | ||||||||
Combined Agreements | |||||||||
Subsidiary, Sale of Stock [Line Items] | |||||||||
Compensation recorded in equity upon the issuance of the shares | $ 51,300 | ||||||||
Common Stock | |||||||||
Subsidiary, Sale of Stock [Line Items] | |||||||||
Issuance of common stock (in shares) | 8,832,565 | 6,571,428 | |||||||
Common Stock | Underwriting Agreement | |||||||||
Subsidiary, Sale of Stock [Line Items] | |||||||||
Issuance of common stock (in shares) | 8,832,565 | 6,571,428 | |||||||
2017 Offering | Common Stock | Underwriting Agreement | |||||||||
Subsidiary, Sale of Stock [Line Items] | |||||||||
Issuance of common stock (in shares) | 5,714,286 | ||||||||
Overallotment | |||||||||
Subsidiary, Sale of Stock [Line Items] | |||||||||
Period to purchase additional shares | 30 days | 30 days | |||||||
Overallotment | Common Stock | Underwriting Agreement | |||||||||
Subsidiary, Sale of Stock [Line Items] | |||||||||
Issuance of common stock (in shares) | 1,152,073 | 857,143 | |||||||
2018 Offering | Common Stock | Underwriting Agreement | |||||||||
Subsidiary, Sale of Stock [Line Items] | |||||||||
Issuance of common stock (in shares) | 7,680,492 | ||||||||
Alexion Share Issuance Agreement | |||||||||
Subsidiary, Sale of Stock [Line Items] | |||||||||
Number of shares issuable (in shares) | 835,834 | ||||||||
Price per share (in dollars per share) | $ 17.95 | ||||||||
Aggregate gross proceeds | $ 15,000 | ||||||||
Lilly Share Issuance Agreement | |||||||||
Subsidiary, Sale of Stock [Line Items] | |||||||||
Number of shares issuable (in shares) | 5,414,185 | ||||||||
Price per share (in dollars per share) | $ 18.47 | ||||||||
Aggregate gross proceeds | $ 100,000 |
REDEEMABLE CONVERTIBLE PREFER_3
REDEEMABLE CONVERTIBLE PREFERRED STOCK - Additional Information (Details) $ / shares in Units, $ in Thousands | Dec. 18, 2017$ / sharesshares | Dec. 13, 2017$ / shares | Oct. 27, 2017 | Oct. 26, 2017 | Apr. 11, 2017USD ($)rate_reduction$ / sharesshares | Mar. 28, 2017director | Mar. 31, 2018 | Jun. 30, 2017USD ($) | Oct. 27, 2017 | Sep. 30, 2017USD ($) | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($)shares | Dec. 31, 2016USD ($) |
Temporary Equity [Line Items] | |||||||||||||
Redeemable convertible preferred stock, issued (in shares) | shares | 700,000 | 700,000 | |||||||||||
Redeemable convertible preferred stock, par value (in dollars per share) | $ / shares | $ 0.0001 | $ 0.0001 | |||||||||||
Redeemable convertible preferred stock, price per share (in dollars per share) | $ / shares | $ 100 | $ 100 | |||||||||||
Proceeds from issuance of common stock to collaboration partners | $ 70,000 | $ 60,412 | $ 0 | $ 0 | |||||||||
Issuance costs related to private placement | $ 800 | $ 703 | 23 | 0 | |||||||||
Existing number of directors | director | 8 | ||||||||||||
Increased number of directors | director | 9 | ||||||||||||
Redeemable convertible preferred stock, terms of conversion | “Conversion Blockers” refers to the beneficial ownership limitations in the Company’s Certificate of Designation of the Redeemable Convertible Preferred, which included (i) a 19.99% blocker provision to comply with Nasdaq Listing Rules, (ii) if so elected by a holder, a 9.99% blocker provision that would have prohibited beneficial ownership of more than 9.99% of the outstanding shares of the Company’s common stock or voting power at any time, and (iii) ownership limitations resulting from applicable regulatory restrictions. | ||||||||||||
Percentage of redeemable convertible preferred stock conversion blocker provision | 19.99% | ||||||||||||
Percentage of redeemable convertible preferred stock ownership limit conversion blocker provision | 9.99% | ||||||||||||
Initial conversion price (in dollars per share) | $ / shares | $ 3.19 | ||||||||||||
Cumulative dividends rate | 8.00% | 12.00% | 8.00% | 12.00% | |||||||||
Convertible share issued (in shares) | shares | 24,206,663 | ||||||||||||
Preferred stock deemed dividend | $ 0 | $ 3,837 | $ 0 | ||||||||||
Percentage of rate reduction | 4.00% | ||||||||||||
Number of interest rate reductions | rate_reduction | 2 | ||||||||||||
REDEEMABLE CONVERTIBLE PREFERRED STOCK | |||||||||||||
Temporary Equity [Line Items] | |||||||||||||
Convertible share issued (in shares) | shares | (755,124) | ||||||||||||
Fair value estimate of dividend declared | $ 1,900 | $ 4,100 | |||||||||||
Deemed dividend related to beneficial conversion feature of redeemable convertible preferred stock | $ 6,100 | ||||||||||||
Pre Funded Warrant | |||||||||||||
Temporary Equity [Line Items] | |||||||||||||
Pre-Funded Warrants issued (in shares) | shares | 0 |
REDEEMABLE CONVERTIBLE PREFER_4
REDEEMABLE CONVERTIBLE PREFERRED STOCK - Schedule of Valuation Assumptions (Details) - REDEEMABLE CONVERTIBLE PREFERRED STOCK | 6 Months Ended | 9 Months Ended |
Jun. 30, 2017$ / shares | Sep. 30, 2017$ / shares | |
Temporary Equity [Line Items] | ||
Price per common share (in dollars per share) | $ 3.17 | $ 5.75 |
Expected term (in years) | ||
Temporary Equity [Line Items] | ||
Expected term (in years) | 6 years 9 months | 6 years 6 months |
Expected volatility | ||
Temporary Equity [Line Items] | ||
Measurement input | 0.700 | 0.730 |
Risk-adjusted discount rate | ||
Temporary Equity [Line Items] | ||
Measurement input | 0.180 | 0.191 |
REDEEMABLE CONVERTIBLE PREFER_5
REDEEMABLE CONVERTIBLE PREFERRED STOCK - Summary of Changes in Redeemable Convertible Preferred (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Increase (Decrease) in Temporary Equity [Roll Forward] | |||
Net proceeds | $ 0 | ||
Dividends accrued at the stated rates | $ 0 | (3,837) | $ 0 |
Conversion of redeemable convertible preferred stock | 0 | 79,361 | 0 |
REDEEMABLE CONVERTIBLE PREFERRED STOCK | |||
Increase (Decrease) in Temporary Equity [Roll Forward] | |||
Balance at January 1, 2017 | $ 0 | 0 | |
Issuance of redeemable convertible preferred shares | 70,000 | ||
Share issuance costs | (750) | ||
Net proceeds | 69,250 | ||
Discount resulting from the BCF at issuance | (6,144) | ||
Accretion of the discount resulting from the BCF (deemed dividend) | 6,144 | ||
Dividends accrued at the stated rates | 5,515 | ||
Fair value in excess of dividends accrued at the stated rates | 3,846 | ||
Accretion of share issuance costs (additional dividends) | 750 | ||
Balance immediately prior to conversion | 79,361 | ||
Conversion of redeemable convertible preferred stock | (79,361) | ||
December 31, 2017 | $ 0 | $ 0 |
STOCK-BASED COMPENSATION - Addi
STOCK-BASED COMPENSATION - Additional Information (Details) - USD ($) $ / shares in Units, $ in Millions | Dec. 11, 2018 | Dec. 10, 2018 | May 31, 2015 | May 31, 2017 | Feb. 28, 2017 | Jun. 30, 2015 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | Dec. 31, 2015 | Dec. 31, 2014 | Mar. 04, 2016 | Jan. 14, 2014 |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Stock options awarded (in shares) | 2,241,350 | ||||||||||||
Number of inducement grants outstanding (in shares) | 7,787,690 | 6,124,096 | |||||||||||
Weighted-average grant date fair value of stock options granted (in dollars per share) | $ 7.64 | $ 2.52 | $ 4.60 | ||||||||||
Unrecognized compensation cost | $ 17.1 | ||||||||||||
Aggregate intrinsic value, outstanding | $ 2.9 | $ 0.1 | $ 0.2 | ||||||||||
Employee Stock Option | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Weighted-average period | 2 years 10 months 24 days | ||||||||||||
2014 Plan | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Maximum number of common stock shares granted (in shares) | 1,900,000 | ||||||||||||
Stock option plan, percentage of outstanding common stock | 4.00% | ||||||||||||
Stock option plan, increased percentage of outstanding common stock | 5.00% | ||||||||||||
Stock option plan, vested rate | 25.00% | ||||||||||||
Stock option plan, vesting period | 36 months | ||||||||||||
Stock option plan, expiration period | 10 years | ||||||||||||
Stock option plan, reserved shares of common stock for future issuance (in shares) | 872,411 | ||||||||||||
2014 Plan | Annual Promotional and Incentive Related Grants | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Stock option plan, vesting period | 48 months | ||||||||||||
Inducement Grants | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Stock options awarded (in shares) | 450,700 | 470,272 | |||||||||||
Number of inducement grants outstanding (in shares) | 130,000 | ||||||||||||
2016 Inducement Plan | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Maximum number of common stock shares granted (in shares) | 250,000 | ||||||||||||
Stock option plan, reserved shares of common stock for future issuance (in shares) | 2,875,000 | ||||||||||||
Stock options awarded (in shares) | 0 | ||||||||||||
Issuable common stock shares (in shares) | 3,275,000 | 2,700,000 | 200,000 | 125,000 |
STOCK-BASED COMPENSATION - Clas
STOCK-BASED COMPENSATION - Classification of Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Stock-based compensation expense | $ 7,888 | $ 7,770 | $ 9,165 |
Research and development expenses | |||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Stock-based compensation expense | 3,062 | 3,536 | 4,467 |
General and administrative expenses | |||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Stock-based compensation expense | $ 4,826 | $ 4,234 | $ 4,698 |
STOCK-BASED COMPENSATION - Sche
STOCK-BASED COMPENSATION - Schedule of Valuation Assumptions (Details) - Employee Stock Option - $ / shares | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Expected dividend yield | 0.00% | 0.00% | 0.00% |
Minimum | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Common stock price (in dollars per share) | $ 9.14 | $ 2.49 | $ 2.94 |
Expected option term (in years) | 5 years 6 months | 5 years 6 months | 5 years 6 months |
Expected volatility | 75.90% | 79.40% | 70.90% |
Risk-free interest rate | 2.30% | 1.90% | 1.20% |
Maximum | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Common stock price (in dollars per share) | $ 15.74 | $ 9.71 | $ 9.09 |
Expected option term (in years) | 6 years 2 months 30 days | 6 years 2 months 30 days | 6 years 2 months 30 days |
Expected volatility | 78.30% | 91.10% | 79.40% |
Risk-free interest rate | 3.00% | 2.20% | 2.00% |
STOCK-BASED COMPENSATION - Summ
STOCK-BASED COMPENSATION - Summary of Activity Under Equity Incentive Plans (Details) $ / shares in Units, $ in Thousands | 12 Months Ended |
Dec. 31, 2018USD ($)$ / sharesshares | |
NUMBER OF OPTIONS | |
Outstanding at beginning of year (in shares) | shares | 6,124,096 |
Granted (in shares) | shares | 2,241,350 |
Exercised (in shares) | shares | (329,934) |
Forfeited/Cancelled (in shares) | shares | (90,026) |
Expired (in shares) | shares | (157,796) |
Outstanding at end of year (in shares) | shares | 7,787,690 |
Exercisable at end of year (in shares) | shares | 4,842,084 |
Vested and expected to vest at end of year (in shares) | shares | 7,567,801 |
WEIGHTED- AVERAGE EXERCISE PRICE | |
Outstanding at beginning of year (in dollars per share) | $ / shares | $ 8.58 |
Granted (in dollars per share) | $ / shares | 11.07 |
Exercised (in dollars per share) | $ / shares | 5.31 |
Forfeited/Cancelled (in dollars per share) | $ / shares | 5.71 |
Expired (in dollars per share) | $ / shares | 14.08 |
Outstanding at end of year (in dollars per share) | $ / shares | 9.36 |
Exercisable at end of year (in dollars per share) | $ / shares | 9.71 |
Vested and expected to vest at end of year (in dollars per share) | $ / shares | $ 9.30 |
WEIGHTED- AVERAGE REMAINING CONTRACTUAL TERM (YEARS) | |
Outstanding at beginning of year | 7 years 3 months 15 days |
Exercisable at end of year | 6 years 4 months 17 days |
Vested and expected to vest at end of year | 7 years 2 months 23 days |
Aggregate intrinsic value, outstanding | $ | $ 24,305 |
Aggregate intrinsic value, exercisable | $ | 16,151 |
Aggregate intrinsic value, vested and expected to vest | $ | $ 23,948 |
STOCK-BASED COMPENSATION - Rest
STOCK-BASED COMPENSATION - Restricted Common Stock (Details) - Restricted Stock - USD ($) $ / shares in Units, $ in Millions | 12 Months Ended | |||
Dec. 31, 2018 | Dec. 31, 2016 | Dec. 31, 2014 | Dec. 31, 2017 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Restricted common stock, shares issued (in shares) | 44,000 | |||
Restricted common stock, shares fully-vested (in shares) | 10,000 | 4,000 | ||
Stock option plan, vesting period | 4 years | |||
Restricted common stock, fair value of shares issued | $ 0.7 | |||
Weighted average grant date fair value per share (in dollars per share) | $ 16.30 | $ 16.30 | $ 16.30 | |
Outstanding shares (in shares) | 0 | |||
Unvested restricted stock outstanding (in shares) | 10,000 | |||
Fair value of vested restricted common stock | $ 0.1 | $ 0.1 |
STOCK-BASED COMPENSATION - Comm
STOCK-BASED COMPENSATION - Common Stock Warrants (Details) - $ / shares | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Class of Warrant or Right [Line Items] | ||
Number of warrants (in shares) | 85,703 | 87,901 |
Exercise price (in dollars per share) | $ 13.80 | |
Warrants received as common stock (in shares) | 45,710 | |
Exercise Price 7.00 | ||
Class of Warrant or Right [Line Items] | ||
Number of warrants (in shares) | 39,993 | |
Exercise price (in dollars per share) | $ 7 | |
Exercise Price 250.00 | ||
Class of Warrant or Right [Line Items] | ||
Number of warrants (in shares) | 2,198 | |
Exercise price (in dollars per share) | $ 250 | |
Remaining life (years) | 1 year 5 months 16 days |
STOCK-BASED COMPENSATION - Empl
STOCK-BASED COMPENSATION - Employee Stock Purchase Plan (Details) - 2014 Employee Stock Purchase Plan | Jan. 28, 2014USD ($)periodshares | Dec. 31, 2018USD ($)$ / sharesshares | Dec. 31, 2017USD ($)$ / sharesshares | Dec. 31, 2016USD ($)$ / sharesshares |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Issuable common stock shares (in shares) | 1,000,000 | |||
Automatic reserve increase equivalent to common stock issued and outstanding percentage | 1.00% | |||
Maximum number of common stock shares granted (in shares) | 2,107,791 | |||
Number of shares available for issuance (in shares) | 1,845,179 | |||
Payroll deduction percentage limit of eligible compensation | 15.00% | |||
Maximum shares per employee (in shares) | 10,000 | |||
Maximum employee contribution amount | $ | $ 25,000 | |||
Offering period | 24 months | |||
Number of purchase periods | period | 4 | |||
Purchase period | 6 months | |||
Stock-based compensation expense related to stock purchase rights | $ | $ 100,000 | $ 400,000 | $ 400,000 | |
Share issued under stock option plan (in shares) | 118,239 | 84,890 | 36,501 | |
Average purchase price of share issued (in dollars per share) | $ / shares | $ 2.61 | $ 2.45 | $ 4.53 | |
Maximum | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Percentage of fair market value of common stock | 85.00% |
401(K) PROFIT SHARING PLAN AN_2
401(K) PROFIT SHARING PLAN AND TRUST (Details) - USD ($) | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Retirement Benefits [Abstract] | |||
Employer matching contribution, percent of match | 300.00% | ||
Employer matching contribution, percent of employees' gross pay | 2.00% | ||
Contributions | $ 600,000 | $ 400,000 | $ 200,000 |
Discretionary costs | $ 0 | $ 0 | $ 0 |
INCOME TAXES - Additional Infor
INCOME TAXES - Additional Information (Details) - USD ($) | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Income Tax [Line Items] | |||
Current income tax expense | $ 0 | $ 0 | |
Deferred income tax expense | 0 | 0 | |
Deferred tax asset, valuation allowance | 17,800,000 | ||
Net operating loss, estimated limitation | $ 97,000,000 | ||
Operating loss carryforward expiration description | Expire starting in 2028 and 2030, respectively. | ||
Tax credit carryforward description | Expire starting in 2028 and 2023, respectively. | ||
Unrecognized tax benefits that would affect income tax expense if recognized | $ 1,600,000 | ||
Accrued penalties or provisions for interest | 0 | ||
Federal | |||
Income Tax [Line Items] | |||
Deferred income tax expense | 0 | $ 0 | $ 0 |
Net operating loss carryforwards | $ 158,700,000 | ||
Operating loss carryforward expiration period | 2028 | ||
Tax credits | $ 2,600,000 | ||
Tax credit carryforward description | 2028 | ||
State | |||
Income Tax [Line Items] | |||
Net operating loss carryforwards | $ 148,200,000 | ||
Operating loss carryforward expiration period | 2030 | ||
Massachusetts | |||
Income Tax [Line Items] | |||
Tax credits | $ 1,400,000 | ||
Tax credit carryforward description | 2023 |
INCOME TAXES - Reconciliation B
INCOME TAXES - Reconciliation Between Income Taxes Computed at the Federal Statutory Income Tax Rate and the Provision for Benefit from Income Taxes (Details) | 12 Months Ended | ||
Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Income Tax Disclosure [Abstract] | |||
Federal statutory rate | 21.00% | 34.00% | 34.00% |
Effect of: | |||
Foreign rate differential | (9.50%) | (17.60%) | (31.40%) |
Tax reform | 0.00% | (29.60%) | 0.00% |
Net operating loss limitation | (23.00%) | 0.00% | 0.00% |
Change in valuation allowance | 10.60% | 13.50% | 0.00% |
Research and development tax credit | 0.50% | 0.60% | (0.70%) |
Stock-based compensation expense | 0.40% | (0.80%) | (0.90%) |
Other | 0.00% | (0.10%) | (1.00%) |
Total | 0.00% | 0.00% | 0.00% |
INCOME TAXES - Deferred Tax Ass
INCOME TAXES - Deferred Tax Assets (Details) - USD ($) $ in Thousands | Dec. 31, 2018 | Dec. 31, 2017 |
Deferred tax assets: | ||
Net operating loss carryforwards | $ 24,739 | $ 32,008 |
Capitalized research and development costs | 516 | 618 |
Research and development credit carryforwards | 3,988 | 3,481 |
Stock-based compensation expense | 7,644 | 6,066 |
Depreciation expense and other costs | 75 | 42 |
Net deferred tax assets | 36,962 | 42,215 |
Valuation allowance | (36,962) | (42,215) |
Net deferred tax assets | $ 0 | $ 0 |
INCOME TAXES - Reconciliation o
INCOME TAXES - Reconciliation of the Gross Unrecognized Tax Benefit (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2018 | Dec. 31, 2017 | |
Reconciliation of Unrecognized Tax Benefits, Excluding Amounts Pertaining to Examined Tax Returns [Roll Forward] | ||
Unrecognized tax benefits at the beginning of the period | $ 1,451 | $ 1,210 |
Additions for current tax positions | 211 | 243 |
Changes for previous tax positions | (31) | (2) |
Unrecognized tax benefits at the end of the period | $ 1,631 | $ 1,451 |
COMMITMENTS AND CONTINGENCIES -
COMMITMENTS AND CONTINGENCIES - Additional Information (Details) | Jul. 11, 2014USD ($)extension | Dec. 31, 2018USD ($) | Dec. 31, 2017USD ($) | Dec. 31, 2016USD ($) |
Loss Contingencies [Line Items] | ||||
Number of options to extend | extension | 1 | |||
Operating lease renewal term | 5 years | |||
Rent expense | $ 1,600,000 | $ 1,600,000 | $ 1,600,000 | |
Cambridge Massachusetts , 2014 Operating Lease Agreement | ||||
Loss Contingencies [Line Items] | ||||
Deferred rent obligation | Dec. 1, 2014 | |||
Operating lease minimum payments | $ 9,600,000 | |||
Operating lease minimum payments term | 6 years | |||
Settled Litigation | ||||
Loss Contingencies [Line Items] | ||||
Outstanding litigation liabilities | $ 0 | $ 0 |
COMMITMENTS AND CONTINGENCIES_2
COMMITMENTS AND CONTINGENCIES - Summary of Future Minimum Lease Payments Payable (Details) $ in Thousands | Dec. 31, 2018USD ($) |
Commitments and Contingencies Disclosure [Abstract] | |
2019 | $ 1,678 |
2020 | 1,581 |
Total | $ 3,259 |
LITIGATION - Additional Informa
LITIGATION - Additional Information (Details) | Oct. 25, 2018USD ($) | Oct. 22, 2018USD ($) | Apr. 20, 2018shares | Apr. 18, 2018USD ($)target | Nov. 30, 2018USD ($) | Dec. 31, 2018USD ($)shares | Dec. 31, 2017USD ($)shares | Dec. 31, 2016USD ($) | Oct. 31, 2018USD ($) | May 31, 2018USD ($) |
Loss Contingencies [Line Items] | ||||||||||
Issuance of common stock (in shares) | shares | 983,208 | |||||||||
Issuance of common stock | $ 107,770,000 | $ 42,779,000 | ||||||||
Litigation expense | 29,132,000 | 9,043,000 | $ 2,916,000 | |||||||
Litigation settlement payable | 10,500,000 | 0 | ||||||||
Interest expense | $ 603,000 | $ 0 | $ 0 | |||||||
Alexion Collaborative Research And License Agreement | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Initial non-refundable upfront payment | $ 22,000,000 | |||||||||
Litigation settlement payable | $ 2,500,000 | |||||||||
Payments for litigation | $ 2,500,000 | |||||||||
Lilly Share Issuance Agreement | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Litigation settlement discount present value | 13,000,000 | |||||||||
Expected proceeds from issuance of common stock | $ 100,000,000 | |||||||||
Alexion Share Issuance Agreement | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Expected proceeds from issuance of common stock | $ 15,000,000 | |||||||||
Common Stock | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Issuance of common stock (in shares) | shares | 8,832,565 | 6,571,428 | ||||||||
Issuance of common stock | $ 1,000 | $ 1,000 | ||||||||
Additional Paid-in Capital | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Issuance of common stock | $ 107,769,000 | 42,778,000 | ||||||||
Alexion Pharmaceuticals | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Cash obligation payable as part of litigation settlement | $ 13,000,000 | |||||||||
Litigation settlement discount present value | $ 8,700,000 | |||||||||
Effective interest rate | 10.00% | |||||||||
Alexion Pharmaceuticals | Common Stock | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Issuance of common stock (in shares) | shares | 983,208 | |||||||||
Alexion Pharmaceuticals | Additional Paid-in Capital | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Litigation expense | $ 3,700,000 | |||||||||
Alnylam | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Cash obligation payable as part of litigation settlement | 10,500,000 | |||||||||
Settled Litigation | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Outstanding litigation liabilities | 0 | $ 0 | ||||||||
Settled Litigation | Alnylam | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Litigation settlement upfront payment payable | $ 2,000,000 | |||||||||
Additional litigation settlement payable amount | $ 13,000,000 | |||||||||
Percentage of additional litigation settlement payable amount | 10.00% | |||||||||
Cash obligation payable as part of litigation settlement | $ 13,000,000 | |||||||||
Number of oligo restrictions target | target | 8 | |||||||||
Litigation expense | $ 24,700,000 | |||||||||
Settled Litigation | Alnylam | Minimum | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Settlement agreement period | 18 months | |||||||||
Settled Litigation | Alnylam | Maximum | ||||||||||
Loss Contingencies [Line Items] | ||||||||||
Settlement agreement period | 4 years |
QUARTERLY FINANCIAL DATA (UNA_3
QUARTERLY FINANCIAL DATA (UNAUDITED) (Details) - USD ($) | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2018 | Sep. 30, 2018 | Jun. 30, 2018 | Mar. 31, 2018 | Dec. 31, 2017 | Sep. 30, 2017 | Jun. 30, 2017 | Mar. 31, 2017 | Dec. 31, 2018 | Dec. 31, 2017 | Dec. 31, 2016 | |
Quarterly Financial Information Disclosure [Abstract] | |||||||||||
Revenue from collaborative arrangements | $ 1,541,000 | $ 1,545,000 | $ 1,545,000 | $ 1,545,000 | $ 1,030,000 | $ 0 | $ 6,176,000 | $ 1,030,000 | $ 0 | ||
Net loss | (18,610,000) | (19,020,000) | (35,644,000) | (15,579,000) | (15,741,000) | (15,033,000) | $ (15,225,000) | $ (14,201,000) | (88,853,000) | (60,200,000) | (59,513,000) |
Net loss attributable to common stockholders | $ (18,610,000) | $ (19,020,000) | $ (35,644,000) | $ (15,579,000) | $ (22,956,000) | $ (19,144,000) | $ (23,991,000) | $ (14,201,000) | $ (88,853,000) | $ (80,292,000) | $ (59,513,000) |
Net loss per share attributable to common stockholders - basic and diluted (in dollars per share) | $ (0.29) | $ (0.35) | $ (0.68) | $ (0.30) | $ (0.91) | $ (0.92) | $ (1.15) | $ (0.68) | $ (1.60) | $ (3.66) | $ (2.87) |
SUBSEQUENT EVENTS (Details)
SUBSEQUENT EVENTS (Details) - Subsequent Event $ in Millions | Jan. 02, 2019USD ($)ft²period | Jan. 04, 2019USD ($)ft² |
Subsequent Event [Line Items] | ||
Area of laboratory and office space (in sqft) | ft² | 80,872 | |
Term of contract | 7 years | |
Number of periods to extend renewal term | period | 2 | |
Lease renewal term | 5 years | |
Annual fixed rent for first 12-month period | $ 3.9 | |
Annual fixed rent on seventh 12-month period | 4.7 | |
Aggregate fixed rent | $ 30.1 | |
Area of office space (in sqft) | ft² | 9,653 | |
Aggregate fixed rent for sublease | $ 0.8 |